PDGFRB

Summary

PDGFRB (platelet derived growth factor receptor beta, HGNC:8804) is a protein-coding gene on chromosome 5q32, encoding Platelet-derived growth factor receptor beta (P09619). Tyrosine-protein kinase that acts as a cell-surface receptor for homodimeric PDGFB and PDGFD and for heterodimers formed by PDGFA and PDGFB, and plays an essential role in the regulation of embryonic development, cell proliferation, survival, differentiation, chemotaxis and migr…. In precision oncology, PDGFRB Overexpression confers sensitivity to Sunitinib in Dermatofibrosarcoma Protuberans (CIViC Level C); 1 further curated variant–drug associations are listed below.

The protein encoded by this gene is a cell surface tyrosine kinase receptor for members of the platelet-derived growth factor family. These growth factors are mitogens for cells of mesenchymal origin. The identity of the growth factor bound to a receptor monomer determines whether the functional receptor is a homodimer (PDGFB or PDGFD) or a heterodimer (PDGFA and PDGFB). This gene is essential for normal development of the cardiovascular system and aids in rearrangement of the actin cytoskeleton. This gene is flanked on chromosome 5 by the genes for granulocyte-macrophage colony-stimulating factor and macrophage-colony stimulating factor receptor; all three genes may be implicated in the 5-q syndrome. A translocation between chromosomes 5 and 12, that fuses this gene to that of the ETV6 gene, results in chronic myeloproliferative disorder with eosinophilia.

Source: NCBI Gene 5159 — RefSeq curated summary.

At a glance

• Gene–disease (curated): acroosteolysis-keloid-like lesions-premature aging syndrome (Definitive, GenCC) — +7 more curated relationships
• GWAS associations: 8
• Clinical variants (ClinVar): 848 total — 20 pathogenic, 5 likely-pathogenic
• Phenotypes (HPO): 179
• Druggable target: yes — 102 molecules with ChEMBL bioactivity
• Precision-oncology evidence (CIViC): 2 curated variant–drug associations
• Cancer driver (intOGen): activating (oncogene-like) across 3 cancer types
• MANE Select transcript: NM_002609

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:8804
Approved symbol	PDGFRB
Name	platelet derived growth factor receptor beta
Location	5q32
Locus type	gene with protein product
Status	Approved
Aliases	JTK12, CD140b, PDGFR1
Ensembl gene	ENSG00000113721
Ensembl biotype	protein_coding
OMIM	173410
Entrez	5159

Gene structure

Transcript identifiers

Ensembl transcripts: 29 — 21 protein_coding, 5 protein_coding_CDS_not_defined, 2 retained_intron, 1 nonsense_mediated_decay

ENST00000261799, ENST00000517488, ENST00000517660, ENST00000517957, ENST00000519575, ENST00000520229, ENST00000520579, ENST00000520851, ENST00000521723, ENST00000522466, ENST00000523456, ENST00000890715, ENST00000890716, ENST00000890717, ENST00000890718, ENST00000890719, ENST00000890720, ENST00000890721, ENST00000890722, ENST00000890723, ENST00000890724, ENST00000890725, ENST00000890726, ENST00000890727, ENST00000951660, ENST00000951661, ENST00000951662, ENST00000951663, ENST00000951664

RefSeq mRNA: 3 — MANE Select: NM_002609 NM_001355016, NM_001355017, NM_002609

CCDS: CCDS4303

Canonical transcript exons

ENST00000261799 — 23 exons

Exon	Start	End
ENSE00001277266	150113839	150115946
ENSE00001277271	150137008	150137053
ENSE00003460521	150126520	150126614
ENSE00003464042	150121204	150121322
ENSE00003469927	150120888	150121010
ENSE00003474441	150125445	150125577
ENSE00003513640	150133586	150133760
ENSE00003517537	150119467	150119566
ENSE00003564035	150121880	150122040
ENSE00003584504	150118747	150118852
ENSE00003600881	150117618	150117850
ENSE00003600936	150124250	150124360
ENSE00003603285	150130539	150130662
ENSE00003607028	150123042	150123201
ENSE00003643730	150124727	150124831
ENSE00003648053	150132750	150132942
ENSE00003652119	150131979	150132094
ENSE00003654111	150135555	150135878
ENSE00003667728	150133881	150134008
ENSE00003682400	150129757	150129968
ENSE00003687411	150120012	150120123
ENSE00003788938	150134750	150135016
ENSE00003845888	150155397	150155845

Expression profiles

Bgee: expression breadth ubiquitous, 270 present calls, max score 99.59.

FANTOM5 (CAGE): breadth ubiquitous, TPM avg 42.3191 / max 697.8902, expressed in 1070 samples.

FANTOM5 promoters (7 alternative TSS)

Promoter ID	TPM avg	Samples expressed
64169	24.2718	961
64170	9.3152	1012
64167	3.9117	737
64165	3.5911	714
64168	0.6448	380
64166	0.5411	286
64164	0.0433	17

Top tissues by expression

296 total, by Bgee expression score (0-100, higher = more expressed):

Tissue	Anatomy ID	Expression score	Quality
stromal cell of endometrium	CL:0002255	99.59	gold quality
right coronary artery	UBERON:0001625	98.97	gold quality
endocervix	UBERON:0000458	98.95	gold quality
popliteal artery	UBERON:0002250	98.83	gold quality
tibial artery	UBERON:0007610	98.83	gold quality
gall bladder	UBERON:0002110	98.69	gold quality
descending thoracic aorta	UBERON:0002345	98.58	gold quality
aorta	UBERON:0000947	98.56	gold quality
right ovary	UBERON:0002118	98.47	gold quality
calcaneal tendon	UBERON:0003701	98.36	gold quality
right lung	UBERON:0002167	98.33	gold quality
left coronary artery	UBERON:0001626	98.32	gold quality
thoracic aorta	UBERON:0001515	98.29	gold quality
ascending aorta	UBERON:0001496	98.27	gold quality
coronary artery	UBERON:0001621	98.27	gold quality
body of uterus	UBERON:0009853	98.27	gold quality
left uterine tube	UBERON:0001303	98.17	gold quality
left ovary	UBERON:0002119	97.93	gold quality
mucosa of stomach	UBERON:0001199	97.90	gold quality
omental fat pad	UBERON:0010414	97.86	gold quality
peritoneum	UBERON:0002358	97.84	gold quality
peripheral nervous system	UBERON:0000010	97.53	gold quality
nerve	UBERON:0001021	97.53	gold quality
tibial nerve	UBERON:0001323	97.53	gold quality
pericardium	UBERON:0002407	97.51	gold quality
subcutaneous adipose tissue	UBERON:0002190	97.46	gold quality
saphenous vein	UBERON:0007318	97.43	gold quality
adipose tissue of abdominal region	UBERON:0007808	97.42	gold quality
apex of heart	UBERON:0002098	97.39	gold quality
ectocervix	UBERON:0012249	97.38	gold quality

Single-cell (SCXA)

Detected in 23 experiment(s), a significant marker in 23.

Experiment	Marker?	Max mean expression
E-ANND-2	yes	2094.99
E-GEOD-134144	yes	1979.16
E-MTAB-11268	yes	1902.31
E-MTAB-6678	yes	1153.93
E-MTAB-9906	yes	934.63
E-MTAB-10662	yes	572.93
E-GEOD-83139	yes	321.81
E-GEOD-81608	yes	270.69
E-HCAD-10	yes	60.17
E-MTAB-8142	yes	55.95
E-MTAB-6701	yes	37.61
E-GEOD-135922	yes	28.98
E-MTAB-8410	yes	26.46
E-HCAD-11	yes	20.66
E-CURD-119	yes	14.91

Regulation

Is transcription factor: no

Upstream regulators (CollecTRI, top): AP1, ATM, FGF9, JUN, JUND, MYC, MYCN, NFKB, PBX1, PPARG, SPI1, TP53, TP73, ZFHX3, ZNF24

miRNA regulators (miRDB)

117 targeting PDGFRB, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-9-5P	100.00	72.28	2361
HSA-MIR-30A-5P	100.00	76.31	3233
HSA-MIR-30B-5P	100.00	76.29	3248
HSA-MIR-30C-5P	100.00	76.29	3248
HSA-MIR-30D-5P	100.00	76.32	3233
HSA-MIR-30E-5P	100.00	76.32	3242
HSA-MIR-574-5P	100.00	66.01	989
HSA-MIR-3134	100.00	66.43	777
HSA-MIR-4692	100.00	67.32	2066
HSA-MIR-548N	99.98	71.94	4170
HSA-MIR-568	99.98	69.86	2084
HSA-MIR-12136	99.98	72.81	5713
HSA-MIR-499A-5P	99.98	70.79	1323
HSA-MIR-6778-3P	99.96	67.29	2693
HSA-MIR-4725-3P	99.96	69.53	2520
HSA-MIR-6780B-5P	99.96	69.60	2562
HSA-MIR-6721-5P	99.93	68.92	2981
HSA-MIR-498-3P	99.91	71.27	1114
HSA-MIR-106A-5P	99.90	73.94	2683
HSA-MIR-4731-5P	99.89	67.23	2537
HSA-MIR-17-5P	99.89	73.83	2665
HSA-MIR-106B-5P	99.88	74.72	2795
HSA-MIR-20A-5P	99.88	74.76	2769
HSA-MIR-20B-5P	99.88	74.01	2621
HSA-MIR-519D-3P	99.88	73.97	2607
HSA-MIR-526B-3P	99.88	74.06	2587
HSA-MIR-93-5P	99.88	73.98	2606
HSA-MIR-4492	99.87	68.25	3611
HSA-MIR-4728-5P	99.85	69.39	4718
HSA-MIR-1321	99.84	65.30	1811

Literature-anchored findings (GeneRIF, showing 40)

• activates phosphatidylinositol 3 (PI3) kinase and requires PI3 kinase to regulate the cell cycle [TEL/platelet-derived growth factor receptor beta] (PMID:11861293)
• With the advent of targeted signal transduction therapy, patients with rearrangement of PDGFRB might be better classified as a distinct subgroup of MPD/MDS. (PMID:11919393)
• Eight out of nine ESFT cell lines were found to express significant levels of beta-PDGFR. Beta-platelet-derived growth factor receptor mediates motility and growth of Ewing’s sarcoma cells. (PMID:12700668)
• overexpressing wild-type PDGFR on exposure to cognate growth factor activate glucose transport, GLUT4 translocation, and the serine-threonine protein kinase Akt/protein kinase B to a degree comparable with that produced in response to insulin. (PMID:12933652)
• YXXM motifs in the PDGFR serve two distinct functions: PI 3-kinase recruitment and lysosomal targeting (PMID:12941951)
• PDGFR is degraded in a process in which NF2 inhibits schwannoma cell growth (PMID:14612918)
• a unique interaction between S1P3 and PDGFR, and demonstration of a specific role for S1P3 in S1P-induced Akt activation (PMID:14657000)
• Both types of PDGF receptors are expressed in follicular keratinocytes, whereas dermal papilla cells only express PDGF receptor beta on the protein level (PMID:14705808)
• results confirm the high frequency of BCR-ABL kinase domain mutations in patients with secondary resistance to imatinib and exclude mutations of the activation loops of KIT, PDGFRA and PDGFRB as causes of resistance in patients without ABL mutations (PMID:14745431)
• ligand-activated KIT and platelet-derived growth factor receptor beta tyrosine kinase receptors are expressed in synovial sarcoma (PMID:14871970)
• allosteric inhibition of the PDGF beta-receptor by its C-terminal tail is one of the mechanisms involved in keeping the receptor inactive in the absence of ligand (PMID:14996833)
• the WW-like domain in the context of TELPDGFbetaR may have both positive and negative regulatory roles in kinase activation. (PMID:15054045)
• GPRK2-mediated PDGFRbeta seryl phosphorylation plays an important role in desensitizing PDGFRbeta; this desensitization involves GPRK2-mediated phosphorylation of PDGFRbeta Ser(1104), with consequent dissociation of the PDGFRbeta from NHERF. (PMID:15271984)
• an activating point mutation results in a deregulated PDGFRbeta with oncogenic predisposition (PMID:15284236)
• In transgenic mice PDGF stimulated glucose transport into skeletal muscle in vitro and in vivo. (PMID:15504957)
• Data demonstrate that type I collagen synergistically enhances platelet-derived growth factor (PDGF)-induced smooth muscle cell proliferation through Src-dependent crosstalk between the alpha2beta1 integrin and the PDGF receptor beta. (PMID:15522237)
• hyperchemotaxis towards PDGFRB is likely to depend in part on phosphorylation of the cytoplasmic domain of TF (PMID:15630487)
• Data show that growth factor-dependent cell survival in vascular smooth muscle cells is mediated only by activation of platelet-derived growth factor-beta receptors and the PI3K/Akt pathway. (PMID:15640155)
• Blocking VEGF and PDGF receptor signaling in cardiac allografts has distinctive effects on inflammation and survival. (PMID:15665766)
• study demonstrates that platelet-derived growth factor receptor alpha and beta, and c-kit protein are expressed in a high percentage of epithelial ovarian cancers (PMID:15791568)
• Western blot showed a high amount of PDGFRalpha and beta proteins in primary stromal cells that could not be detected in prostatic cancer cells. (PMID:15862965)
• PDGFR-beta activation promotes its association with the low density lipoprotein receptor-related protein (PMID:15944146)
• PDGFR-dependent cell detachment is induced by S1P via inhibition of beta1 integrin in HEK293 cells (PMID:15987639)
• Resultrs suggest that activated platelet-derived growth factor receptor-beta (PDGFRbeta) phosphorylates GRK2 tyrosyl residues and thereby activates GRK2, which then serine-phosphorylates and desensitizes the PDGFRbeta. (PMID:15994317)
• alpha-PDGFR expression might identify prostate cancer cells with the highest propensity to metastasize to the skeleton (PMID:16007172)
• PDGF-BB/PDGF-R interaction is vital in nicotine’s mitogenic actions on human aortic smooth muscle cells (PMID:16149045)
• Serum starvation significantly increases PDGFbeta-R mRNA and protein expression in smooth muscle cells. PDGF-BB stimulates cell survival but not proliferation in serum-starved SMCs (PMID:16407661)
• As such, caveolae and caveolin-1 coordinate PDGF receptor signaling, leading to myocyte proliferation, and inhibit constitutive activity of p42/p44 MAPK to sustain cell quiescence. (PMID:16617096)
• The involvement of the PDGFRbeta gene in SFT is compatible with a pericytic derivation, also supporting a possible role of this tyrosine kinase in malignant transformation and in the adoption of novel molecular therapies. (PMID:16899864)
• In midgut carcinoid tumor stroma PDGFRalpha was expressed in 35%, PDGFRbeta in 94% and EGFR in 9%. (PMID:17047316)
• Alix inhibits down-regulation of PDGFRbeta by modulating the interaction between c-Cbl and the receptor, thereby affecting the ubiquitination of the receptor (PMID:17082185)
• The PDGFRB was highly expressed and phosphorylated with the absence of gain-of-function mutations and the presence of the cognate ligands, strongly support the hypothesis that the activation mechanism is the autocrine/paracrine loop in chordoma. (PMID:17145809)
• PDGFRbeta and FLT3 mutants are inhibited by sorafenib (PMID:17229632)
• numerous partner genes fuse to PDGFRB in BCR-ABL negative chronic myeloproliferative disorders (PMID:17296564)
• no activating mutations in the PDGFRB gene were found in children with Ewing sarcoma (PMID:17298867)
• Gene rearrangement found in a case of underlying chronic myelomonocytic leukaemia in Noonan Syndrome. (PMID:17301821)
• these observations indicate that an IE and/or E human cytomegalovirus protein(s) downregulates the expression of PDGFR-alpha and -beta in smooth muscle cells (PMID:17344284)
• Stimulatory antibodies to PDGFR-A&-B were found selectively in all patients with extensive cGVHD but in none of the patients without cGVHD. Higher levels were detected in patients with generalized skin involvement and/or lung fibrosis. (PMID:17363728)
• association of the PDGFRbeta with lipid raft microdomains renders it susceptible to LXA(4)-mediated dephosphorylation by possible reactivation of oxidatively inactivated SHP-2. (PMID:17403678)
• The presence of PDGFB and PDGFRB mRNAs was demonstrated in 26 and 21 of 26 cases dermatofibrosarcoma protuberans. The fusion protein, which is processed by the COL1A1-PDGFB transcripts, can serve as a functional ligand for PDGFRB (PMID:17431412)

Cross-species orthologs

3 orthologs

Organism	Symbol	Gene ID
danio_rerio	pdgfrb	ENSDARG00000100897
mus_musculus	Pdgfrb	ENSMUSG00000024620
rattus_norvegicus	Pdgfrb	ENSRNOG00000018461

Paralogs (53): INSRR (ENSG00000027644), MUSK (ENSG00000030304), FLT4 (ENSG00000037280), EPHA3 (ENSG00000044524), ROS1 (ENSG00000047936), LTK (ENSG00000062524), ERBB3 (ENSG00000065361), TIE1 (ENSG00000066056), FGFR2 (ENSG00000066468), FGFR3 (ENSG00000068078), EPHA8 (ENSG00000070886), FGFR1 (ENSG00000077782), EPHA6 (ENSG00000080224), TYRO3 (ENSG00000092445), FLT1 (ENSG00000102755), MET (ENSG00000105976), EPHB6 (ENSG00000106123), EPHA4 (ENSG00000116106), TEK (ENSG00000120156), FLT3 (ENSG00000122025), KDR (ENSG00000128052), EPHB2 (ENSG00000133216), PDGFRA (ENSG00000134853), EPHA7 (ENSG00000135333), IGF1R (ENSG00000140443), NTRK3 (ENSG00000140538), ERBB2 (ENSG00000141736), EPHA2 (ENSG00000142627), EPHA5 (ENSG00000145242), EGFR (ENSG00000146648), EPHA1 (ENSG00000146904), NTRK2 (ENSG00000148053), MERTK (ENSG00000153208), EPHB1 (ENSG00000154928), KIT (ENSG00000157404), FGFR4 (ENSG00000160867), DDR2 (ENSG00000162733), RYK (ENSG00000163785), MST1R (ENSG00000164078), LMTK2 (ENSG00000164715)

Protein

Protein identifiers

Platelet-derived growth factor receptor beta — P09619 (reviewed: P09619)

Alternative names: Beta platelet-derived growth factor receptor, Beta-type platelet-derived growth factor receptor, CD140 antigen-like family member B, Platelet-derived growth factor receptor 1

All UniProt accessions (4): P09619, E5RH16, E5RII0, E5RJ14

UniProt curated annotations — full annotation on UniProt →

Function. Tyrosine-protein kinase that acts as a cell-surface receptor for homodimeric PDGFB and PDGFD and for heterodimers formed by PDGFA and PDGFB, and plays an essential role in the regulation of embryonic development, cell proliferation, survival, differentiation, chemotaxis and migration. Plays an essential role in blood vessel development by promoting proliferation, migration and recruitment of pericytes and smooth muscle cells to endothelial cells. Plays a role in the migration of vascular smooth muscle cells and the formation of neointima at vascular injury sites. Required for normal development of the cardiovascular system. Required for normal recruitment of pericytes (mesangial cells) in the kidney glomerulus, and for normal formation of a branched network of capillaries in kidney glomeruli. Promotes rearrangement of the actin cytoskeleton and the formation of membrane ruffles. Binding of its cognate ligands - homodimeric PDGFB, heterodimers formed by PDGFA and PDGFB or homodimeric PDGFD -leads to the activation of several signaling cascades; the response depends on the nature of the bound ligand and is modulated by the formation of heterodimers between PDGFRA and PDGFRB. Phosphorylates PLCG1, PIK3R1, PTPN11, RASA1/GAP, CBL, SHC1 and NCK1. Activation of PLCG1 leads to the production of the cellular signaling molecules diacylglycerol and inositol 1,4,5-trisphosphate, mobilization of cytosolic Ca(2+) and the activation of protein kinase C. Phosphorylation of PIK3R1, the regulatory subunit of phosphatidylinositol 3-kinase, leads to the activation of the AKT1 signaling pathway. Phosphorylation of SHC1, or of the C-terminus of PTPN11, creates a binding site for GRB2, resulting in the activation of HRAS, RAF1 and down-stream MAP kinases, including MAPK1/ERK2 and/or MAPK3/ERK1. Promotes phosphorylation and activation of SRC family kinases. Promotes phosphorylation of PDCD6IP/ALIX and STAM. Receptor signaling is down-regulated by protein phosphatases that dephosphorylate the receptor and its down-stream effectors, and by rapid internalization of the activated receptor.

Subunit / interactions. Interacts with homodimeric PDGFB and PDGFD, and with heterodimers formed by PDGFA and PDGFB. May also interact with homodimeric PDGFC. Monomer in the absence of bound ligand. Interaction with homodimeric PDGFB, heterodimers formed by PDGFA and PDGFB or homodimeric PDGFD, leads to receptor dimerization, where both PDGFRA homodimers and heterodimers with PDGFRB are observed. Interacts with SH2B2/APS. Interacts directly (tyrosine phosphorylated) with SHB. Interacts (tyrosine phosphorylated) with PIK3R1 and RASA1. Interacts (tyrosine phosphorylated) with CBL. Interacts (tyrosine phosphorylated) with SRC and SRC family kinases. Interacts (tyrosine phosphorylated) with PIK3C2B, maybe indirectly. Interacts (tyrosine phosphorylated) with SHC1, GRB7, GRB10 and NCK1. Interaction with GRB2 is mediated by SHC1. Interacts (via C-terminus) with NHERF1.

Subcellular location. Cell membrane. Cytoplasmic vesicle. Lysosome lumen.

Post-translational modifications. Autophosphorylated on tyrosine residues upon ligand binding. Autophosphorylation occurs in trans, i.e. one subunit of the dimeric receptor phosphorylates tyrosine residues on the other subunit. Phosphorylation at Tyr-579, and to a lesser degree, at Tyr-581, is important for interaction with SRC family kinases. Phosphorylation at Tyr-740 and Tyr-751 is important for interaction with PIK3R1. Phosphorylation at Tyr-751 is important for interaction with NCK1. Phosphorylation at Tyr-771 and Tyr-857 is important for interaction with RASA1/GAP. Phosphorylation at Tyr-857 is important for efficient phosphorylation of PLCG1 and PTPN11, resulting in increased phosphorylation of AKT1, MAPK1/ERK2 and/or MAPK3/ERK1, PDCD6IP/ALIX and STAM, and in increased cell proliferation. Phosphorylation at Tyr-1009 is important for interaction with PTPN11. Phosphorylation at Tyr-1009 and Tyr-1021 is important for interaction with PLCG1. Phosphorylation at Tyr-1021 is important for interaction with CBL; PLCG1 and CBL compete for the same binding site. Dephosphorylated by PTPRJ at Tyr-751, Tyr-857, Tyr-1009 and Tyr-1021. Dephosphorylated by PTPN2 at Tyr-579 and Tyr-1021. N-glycosylated. Ubiquitinated. After autophosphorylation, the receptor is polyubiquitinated, leading to its degradation.

Disease relevance. A chromosomal aberration involving PDGFRB is found in a form of chronic myelomonocytic leukemia (CMML). Translocation t(5;12)(q33;p13) with EVT6/TEL. It is characterized by abnormal clonal myeloid proliferation and by progression to acute myelogenous leukemia (AML). Myeloproliferative disorder chronic with eosinophilia (MPE) [MIM:131440] A hematologic disorder characterized by malignant eosinophils proliferation. The gene represented in this entry may be involved in disease pathogenesis. Chromosomal aberrations involving PDGFRB have been found in many instances of chronic myeloproliferative disorder with eosinophilia. Translocation t(5;12) with ETV6 on chromosome 12 creating an PDGFRB-ETV6 fusion protein. Translocation t(5;15)(q33;q22) with TP53BP1 creating a PDGFRB-TP53BP1 fusion protein. Translocation t(1;5)(q23;q33) that forms a PDE4DIP-PDGFRB fusion protein. Translocation t(5;6)(q33-34;q23) with CEP85L that fuses the 5’-end of CEP85L (isoform 4) to the 3’-end of PDGFRB. Leukemia, acute myelogenous (AML) [MIM:601626] A subtype of acute leukemia, a cancer of the white blood cells. AML is a malignant disease of bone marrow characterized by maturational arrest of hematopoietic precursors at an early stage of development. Clonal expansion of myeloid blasts occurs in bone marrow, blood, and other tissue. Myelogenous leukemias develop from changes in cells that normally produce neutrophils, basophils, eosinophils and monocytes. The gene represented in this entry may be involved in disease pathogenesis. A chromosomal aberration involving PDGFRB has been found in a patient with AML. Translocation t(5;14)(q33;q32) with TRIP11. Leukemia, juvenile myelomonocytic (JMML) [MIM:607785] An aggressive pediatric myelodysplastic syndrome/myeloproliferative disorder characterized by malignant transformation in the hematopoietic stem cell compartment with proliferation of differentiated progeny. Patients have splenomegaly, enlarged lymph nodes, rashes, and hemorrhages. The gene represented in this entry may be involved in disease pathogenesis. A chromosomal aberration involving PDGFRB has been found in a patient with JMML. Translocation t(5;17)(q33;p11.2) with SPECC1. Basal ganglia calcification, idiopathic, 4 (IBGC4) [MIM:615007] A form of basal ganglia calcification, an autosomal dominant condition characterized by symmetric calcification in the basal ganglia and other brain regions. Affected individuals can either be asymptomatic or show a wide spectrum of neuropsychiatric symptoms, including parkinsonism, dystonia, tremor, ataxia, dementia, psychosis, seizures, and chronic headache. Serum levels of calcium, phosphate, alkaline phosphatase and parathyroid hormone are normal. The neuropathological hallmark of the disease is vascular and pericapillary calcification, mainly of calcium phosphate, in the affected brain areas. The disease is caused by variants affecting the gene represented in this entry. Myofibromatosis, infantile 1 (IMF1) [MIM:228550] A rare mesenchymal disorder characterized by the development of benign tumors in the skin, striated muscles, bones, and, more rarely, visceral organs. Subcutaneous or soft tissue nodules commonly involve the skin of the head, neck, and trunk. Skeletal and muscular lesions occur in about half of the patients. Lesions may be solitary or multicentric, and they may be present at birth or become apparent in early infancy or occasionally in adult life. Visceral lesions are associated with high morbidity and mortality. The disease is caused by variants affecting the gene represented in this entry. Kosaki overgrowth syndrome (KOGS) [MIM:616592] A syndrome characterized by somatic overgrowth, distinctive facial features, hyperelastic and fragile skin, and progressive neurologic deterioration with white matter lesions on brain imaging. The disease is caused by variants affecting the gene represented in this entry. Premature aging syndrome, Penttinen type (PENTT) [MIM:601812] An autosomal dominant syndrome characterized by a prematurely aged appearance with lipoatrophy, epidermal and dermal atrophy along with hypertrophic lesions that resemble scars, thin hair, proptosis, underdeveloped cheekbones, and marked acro-osteolysis. The disease is caused by variants affecting the gene represented in this entry. Ocular pterygium-digital keloid dysplasia syndrome (OPDKD) [MIM:621091] An autosomal dominant disorder that presents in childhood with aggressive ingrowth of vascularized connective tissue on the cornea, ultimately leading to loss of vision. Later, affected individuals develop keloids on digits after minor trauma, but are otherwise healthy. The disease may be caused by variants affecting the gene represented in this entry.

Activity regulation. Present in an inactive conformation in the absence of bound ligand. Binding of PDGFB and/or PDGFD leads to dimerization and activation by autophosphorylation on tyrosine residues. Inhibited by imatinib.

Similarity. Belongs to the protein kinase superfamily. Tyr protein kinase family. CSF-1/PDGF receptor subfamily.

Isoforms (2)

UniProt ID	Names	Canonical?
P09619-1	1	yes
P09619-2	2

RefSeq proteins (3): NP_001341945, NP_001341946, NP_002600* (*=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR000719	Prot_kinase_dom	Domain
IPR001245	Ser-Thr/Tyr_kinase_cat_dom	Domain
IPR001824	Tyr_kinase_rcpt_3_CS	Conserved_site
IPR003598	Ig_sub2	Domain
IPR003599	Ig_sub	Domain
IPR007110	Ig-like_dom	Domain
IPR008266	Tyr_kinase_AS	Active_site
IPR011009	Kinase-like_dom_sf	Homologous_superfamily
IPR013151	Immunoglobulin_dom	Domain
IPR013783	Ig-like_fold	Homologous_superfamily
IPR017441	Protein_kinase_ATP_BS	Binding_site
IPR020635	Tyr_kinase_cat_dom	Domain
IPR027288	PGFRB	Family
IPR036179	Ig-like_dom_sf	Homologous_superfamily
IPR050122	RTK	Family

Pfam: PF00047, PF07714, PF13927, PF25305

Enzyme classification (BRENDA):

• EC 2.7.10.1 — receptor protein-tyrosine kinase (BRENDA: 44 organisms, 214 substrates, 574 inhibitors, 11 Km, 0 kcat entries)

Substrate kinetics (BRENDA)

4 substrates with measured Km, best-characterized 4. Km ranges are aggregated across organisms/conditions.

Substrate	Km (mM)	Measurements
ATP	0.0011–0.129	4
AC-DYFE-6-CHLORO-W-NHME	0.0051	1
AC-DYFGW-NHME	0.07	1
YFEW	0.232	1

Catalyzed reactions (Rhea), 1 shown:

• L-tyrosyl-[protein] + ATP = O-phospho-L-tyrosyl-[protein] + ADP + H(+) (RHEA:10596)

UniProt features (113 total): strand 23, sequence variant 17, modified residue 16, mutagenesis site 13, glycosylation site 11, domain 6, disulfide bond 4, turn 3, helix 3, site 3, compositionally biased region 2, binding site 2, topological domain 2, splice variant 2, signal peptide 1, chain 1, region of interest 1, active site 1, transmembrane region 1, sequence conflict 1

Structure

Experimental structures (PDB)

8 structures.

PDB	Method	Resolution (Å)
1SHA	X-RAY DIFFRACTION	1.5
1H9O	X-RAY DIFFRACTION	1.79
1GQ5	X-RAY DIFFRACTION	2.2
3MJG	X-RAY DIFFRACTION	2.3
2IUI	X-RAY DIFFRACTION	2.4
2L6W	SOLUTION NMR
2PLD	SOLUTION NMR
2PLE	SOLUTION NMR

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-P09619-F1	72.96	0.30

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Catalytic / active sites (4): 826 (proton acceptor); 527–528 (breakpoint for insertion to form pde4dip-pdgfrb fusion protein); 527–528 (breakpoint for translocation to form trip11-pdgfrb); 558–559 (breakpoint for translocation to form the cep85l-pdgfrb fusion protein)

Ligand- & substrate-binding residues (2): 606–614; 634

Post-translational modifications (16): 562, 579, 581, 686, 716, 740, 751, 763, 771, 775, 778, 857, 934, 970, 1009, 1021

Disulfide bonds (4): 54–100, 149–190, 235–291, 436–508

Glycosylation sites (11): 45, 89, 103, 215, 230, 292, 307, 354, 371, 468, 479

Mutagenesis-validated functional residues (13):

Position	Phenotype
579	loss of kinase activity; when associated with f-581. strongly reduces interaction with src family kinases. no effect on
581	loss of kinase activity; when associated with f-579. no effect on interaction with grb10.
634	loss of kinase activity. abolishes interaction with rasa1. no effect on phosphatidylinositol 3-kinase activity.
716	no effect neither on interaction with grb10 and rasa1 nor on phosphatidylinositol 3-kinase activity.
740	strongly reduces up-regulation of cell proliferation; when associated with f-751. strongly decreases phosphatidylinosito
751	strongly reduces up-regulation of cell proliferation; when associated with f-740. abolishes phosphatidylinositol 3-kinas
763	no effect on interaction with rasa1 and on phosphatidylinositol 3-kinase activity.
771	loss of interaction with grb10. abolishes interaction with rasa1. no effect on phosphatidylinositol 3-kinase activity.
775	no effect on interaction with rasa1 and on phosphatidylinositol 3-kinase activity.
778	strongly reduces expression levels.
857	reduces kinase activity. no effect on interaction with grb10. abolishes interaction with rasa1. no effect on phosphatidy
1009	no effect on interaction with grb10. abolishes interaction with plcg1; when associated with f-1021.
1021	strongly reduces up-regulation of cell proliferation. abolishes interaction with plcg1; when associated with f-1009. no

Function

Pathways and Gene Ontology

Reactome pathways

6 pathways

ID	Pathway
R-HSA-1257604	PIP3 activates AKT signaling
R-HSA-186763	Downstream signal transduction
R-HSA-186797	Signaling by PDGF
R-HSA-2219530	Constitutive Signaling by Aberrant PI3K in Cancer
R-HSA-5673001	RAF/MAP kinase cascade
R-HSA-6811558	PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling

MSigDB gene sets: 843 (showing top): PID_SHP2_PATHWAY, GOBP_PLATELET_DERIVED_GROWTH_FACTOR_RECEPTOR_SIGNALING_PATHWAY, GOBP_POSITIVE_REGULATION_OF_CALCIUM_ION_TRANSPORT, TGGTGCT_MIR29A_MIR29B_MIR29C, RNGTGGGC_UNKNOWN, GOBP_POSITIVE_REGULATION_OF_MITOTIC_NUCLEAR_DIVISION, GOBP_CELL_MIGRATION_INVOLVED_IN_HEART_DEVELOPMENT, PID_S1P_S1P1_PATHWAY, GOBP_MUSCLE_TISSUE_DEVELOPMENT, CHIBA_RESPONSE_TO_TSA_UP, GOBP_VASCULAR_ENDOTHELIAL_GROWTH_FACTOR_SIGNALING_PATHWAY, GOBP_POSITIVE_REGULATION_OF_DNA_BIOSYNTHETIC_PROCESS, GOBP_REGULATION_OF_CALCIUM_MEDIATED_SIGNALING, GOBP_METANEPHROS_DEVELOPMENT, GOBP_CORONARY_VASCULATURE_DEVELOPMENT

GO Biological Process (38): angiogenesis (GO:0001525), signal transduction (GO:0007165), cell surface receptor protein tyrosine kinase signaling pathway (GO:0007169), positive regulation of cell population proliferation (GO:0008284), smooth muscle adaptation (GO:0014805), positive regulation of smooth muscle cell migration (GO:0014911), peptidyl-tyrosine phosphorylation (GO:0018108), positive regulation of cell migration (GO:0030335), regulation of actin cytoskeleton organization (GO:0032956), cell migration involved in vasculogenesis (GO:0035441), platelet-derived growth factor receptor-beta signaling pathway (GO:0035791), positive regulation of metanephric mesenchymal cell migration by platelet-derived growth factor receptor-beta signaling pathway (GO:0035793), aorta morphogenesis (GO:0035909), positive regulation of cell proliferation by VEGF-activated platelet derived growth factor receptor signaling pathway (GO:0038091), positive regulation of MAP kinase activity (GO:0043406), positive regulation of mitotic nuclear division (GO:0045840), protein autophosphorylation (GO:0046777), platelet-derived growth factor receptor signaling pathway (GO:0048008), positive regulation of smooth muscle cell proliferation (GO:0048661), positive regulation of calcium-mediated signaling (GO:0050850), positive regulation of chemotaxis (GO:0050921), positive regulation of phosphatidylinositol 3-kinase/protein kinase B signal transduction (GO:0051897), cardiac myofibril assembly (GO:0055003), cell chemotaxis (GO:0060326), cell migration involved in coronary angiogenesis (GO:0060981), retina vasculature development in camera-type eye (GO:0061298), positive regulation of ERK1 and ERK2 cascade (GO:0070374), smooth muscle cell chemotaxis (GO:0071670), metanephric glomerular mesangial cell proliferation involved in metanephros development (GO:0072262), metanephric glomerular capillary formation (GO:0072277), positive regulation of calcium ion import (GO:0090280), positive regulation of reactive oxygen species metabolic process (GO:2000379), positive regulation of DNA biosynthetic process (GO:2000573), protein phosphorylation (GO:0006468), chemotaxis (GO:0006935), G protein-coupled receptor signaling pathway (GO:0007186), cell migration (GO:0016477), cellular response to platelet-derived growth factor stimulus (GO:0036120)

GO Molecular Function (19): protein kinase activity (GO:0004672), protein tyrosine kinase activity (GO:0004713), platelet activating factor receptor activity (GO:0004992), platelet-derived growth factor receptor activity (GO:0005017), platelet-derived growth factor beta-receptor activity (GO:0005019), GTPase activator activity (GO:0005096), signaling receptor binding (GO:0005102), platelet-derived growth factor receptor binding (GO:0005161), ATP binding (GO:0005524), enzyme binding (GO:0019899), protein kinase binding (GO:0019901), vascular endothelial growth factor binding (GO:0038085), platelet-derived growth factor binding (GO:0048407), phospholipase C activator activity (GO:0160185), nucleotide binding (GO:0000166), transmembrane receptor protein tyrosine kinase activity (GO:0004714), protein binding (GO:0005515), kinase activity (GO:0016301), transferase activity (GO:0016740)

GO Cellular Component (11): nucleus (GO:0005634), cytoplasm (GO:0005737), Golgi apparatus (GO:0005794), plasma membrane (GO:0005886), focal adhesion (GO:0005925), membrane (GO:0016020), apical plasma membrane (GO:0016324), cytoplasmic vesicle (GO:0031410), lysosomal lumen (GO:0043202), signaling receptor complex (GO:0043235), lysosome (GO:0005764)

Reactome top-level categories

Rollup of top-6 pathways:

Category	Pathways
Intracellular signaling by second messengers	1
Signaling by PDGF	1
Signaling by Receptor Tyrosine Kinases	1
PI3K/AKT Signaling in Cancer	1
MAPK1/MAPK3 signaling	1
Negative regulation of the PI3K/AKT network	1

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
cell population proliferation	2
protein phosphorylation	2
cell migration	2
platelet-derived growth factor receptor signaling pathway	2
positive regulation of cell population proliferation	2
protein binding	2
growth factor binding	2
intracellular membrane-bounded organelle	2
cellular anatomical structure	2
cytoplasm	2
blood vessel morphogenesis	1
anatomical structure formation involved in morphogenesis	1
cell communication	1
cellular process	1
signaling	1
regulation of cellular process	1
cellular response to stimulus	1
enzyme-linked receptor protein signaling pathway	1
regulation of cell population proliferation	1
positive regulation of cellular process	1
muscle adaptation	1
smooth muscle cell migration	1
regulation of smooth muscle cell migration	1
positive regulation of cell migration	1
peptidyl-tyrosine modification	1
regulation of cell migration	1
positive regulation of cell motility	1
actin cytoskeleton organization	1
regulation of actin filament-based process	1
regulation of cytoskeleton organization	1
vasculogenesis	1
metanephric mesenchymal cell migration	1
regulation of metanephric mesenchymal cell migration by platelet-derived growth factor receptor-beta signaling pathway	1
positive regulation of metanephric mesenchymal cell migration	1
aorta development	1
artery morphogenesis	1
VEGF-activated platelet-derived growth factor receptor signaling pathway	1
MAP kinase activity	1
regulation of MAP kinase activity	1
positive regulation of MAPK cascade	1

Protein interactions and networks

STRING

4460 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
PDGFRB	PDGFB	P01127	999
PDGFRB	PDGFA	P04085	997
PDGFRB	PDGFD	Q9GZP0	997
PDGFRB	PDGFC	Q9NRA1	985
PDGFRB	KDR	P35968	979
PDGFRB	SRC	P12931	972
PDGFRB	EGFR	P00533	971
PDGFRB	GRB2	P29354	955
PDGFRB	PTPN11	Q06124	943
PDGFRB	PLCG1	P19174	914
PDGFRB	EGF	P01133	901
PDGFRB	PIK3R1	P27986	900
PDGFRB	ETV6	P41212	855
PDGFRB	RASA1	P20936	850
PDGFRB	FGF2	P09038	846

IntAct

359 interactions, top by confidence:

A	B	Type	Score
PDGFRB	PDGFB	psi-mi:“MI:0915”(physical association)	0.900
PDGFB	PDGFRB	psi-mi:“MI:0407”(direct interaction)	0.900
PDGFRB	PTPN11	psi-mi:“MI:2364”(proximity)	0.900
PLCG1	PDGFRB	psi-mi:“MI:0407”(direct interaction)	0.850
PDGFRB	PLCG1	psi-mi:“MI:2364”(proximity)	0.850
TSPAN15	ADAM10	psi-mi:“MI:0914”(association)	0.840
TSPAN5	ADAM10	psi-mi:“MI:0914”(association)	0.800
PIK3R3	PIK3CD	psi-mi:“MI:0914”(association)	0.800
PDGFRB	PDGFRA	psi-mi:“MI:0914”(association)	0.770
CD9	ADAM10	psi-mi:“MI:0914”(association)	0.750
PDGFRB	NHERF1	psi-mi:“MI:2364”(proximity)	0.730
PDGFRB	KLHL12	psi-mi:“MI:0915”(physical association)	0.720
KLHL12	PDGFRB	psi-mi:“MI:0915”(physical association)	0.720
PDGFRB	PTPN12	psi-mi:“MI:0915”(physical association)	0.700
PTEN	PDGFRB	psi-mi:“MI:2364”(proximity)	0.670
PDGFRB	NHERF2	psi-mi:“MI:0407”(direct interaction)	0.620
PDGFRB	PIK3R2	psi-mi:“MI:0914”(association)	0.610
GOPC	PDGFRB	psi-mi:“MI:0407”(direct interaction)	0.570
PDGFRB	TRIP6	psi-mi:“MI:0915”(physical association)	0.560
TRIP6	PDGFRB	psi-mi:“MI:0915”(physical association)	0.560
PDGFRB	KRT31	psi-mi:“MI:0915”(physical association)	0.560
KRT34	PDGFRB	psi-mi:“MI:0915”(physical association)	0.560
PDGFRB	CEP76	psi-mi:“MI:0915”(physical association)	0.560
PDGFRB	KRTAP10-9	psi-mi:“MI:0915”(physical association)	0.560

BioGRID (292): TRIP6 (Two-hybrid), KLHL12 (Two-hybrid), PDGFB (Affinity Capture-Western), PDGFB (Reconstituted Complex), KRTAP4-12 (Two-hybrid), PDGFRB (Affinity Capture-Western), TNK2 (Biochemical Activity), PDGFRB (Affinity Capture-MS), MYO1E (Affinity Capture-MS), BCLAF1 (Affinity Capture-MS), ZZEF1 (Affinity Capture-MS), FGD6 (Affinity Capture-MS), FOXK1 (Affinity Capture-MS), PIK3R3 (Co-localization), PTEN (Co-localization)

ESM2 similar proteins: A0JM41, A2VD98, A6QQC6, A8MVW5, B0CLX4, B6ZK76, B6ZK77, O60487, O70255, O88324, O88775, O95976, P01832, P03228, P06907, P08920, P08921, P09619, P0C6B7, P0C6N0, P0CW72, P10522, P20938, P21995, P25189, P27573, P37301, P37998, P59823, P59824, P86176, Q01151, Q4VAH7, Q5EAB0, Q5R804, Q640U3, Q6PCB8, Q6WEB5, Q80UL9, Q86XK7

Diamond homologs: D2IYS2, G3V9H8, O08775, O42127, O73791, O73798, O97799, P00529, P00545, P04048, P05532, P05622, P07333, P07949, P09581, P09619, P10721, P11362, P13369, P16092, P16234, P17948, P18460, P18461, P20786, P21802, P21803, P21804, P22182, P22455, P22607, P23049, P26618, P26619, P33497, P35546, P35590, P35916, P35917, P35918

SIGNOR signaling

95 interactions.

A	Effect	B	Mechanism
PDGFB	up-regulates	PDGFRB	binding
PDGFRB	up-regulates	RASA1	binding
sunitinib	down-regulates	PDGFRB	“chemical inhibition”
PDGFRB	up-regulates	PTK2	phosphorylation
PDGFRB	up-regulates	PDGFRB	phosphorylation
axitinib	down-regulates	PDGFRB	“chemical inhibition”
PTPN1	down-regulates	PDGFRB	dephosphorylation
ABL1	down-regulates	PDGFRB	phosphorylation
PDGFRB	up-regulates	CRK	binding
Crenolanib	down-regulates	PDGFRB	“chemical inhibition”
linifanib	down-regulates	PDGFRB	“chemical inhibition”
MK-2461	down-regulates	PDGFRB	“chemical inhibition”
Telatinib	down-regulates	PDGFRB	“chemical inhibition”
orantinib	down-regulates	PDGFRB	“chemical inhibition”
PDGFRB	unknown	PDGFRB	phosphorylation
PDGFRB	“up-regulates activity”	SRC	phosphorylation
ACP1	“down-regulates activity”	PDGFRB	dephosphorylation
PTPRJ	“down-regulates activity”	PDGFRB	dephosphorylation
PDGFRB	“up-regulates activity”	PDGFRB	phosphorylation

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 157 IntAct physical interaction partners (hypergeometric vs the genome-wide background, BH-FDR, gene-set size 15–500, ranked by fold). A functional readout of the neighbourhood — distinct from this gene’s own memberships above, and biased toward well-studied / hub proteins, so read it as themes rather than proof.

Reactome pathways:

Pathway	Partners	Fold	FDR
Downstream signal transduction	9	31.1×	5e-09
Ras activation upon Ca2+ influx through NMDA receptor	5	25.9×	1e-04
Unblocking of NMDA receptors, glutamate binding and activation	5	24.7×	1e-04
Negative regulation of NMDA receptor-mediated neuronal transmission	5	24.7×	1e-04
Interleukin-37 signaling	5	23.6×	1e-04
Signaling by phosphorylated juxtamembrane, extracellular and kinase domain KIT mutants	5	23.6×	1e-04
Long-term potentiation	5	21.6×	2e-04
Growth hormone receptor signaling	5	21.6×	2e-04

GO biological processes:

GO term	Partners	Fold	FDR
peptidyl-tyrosine dephosphorylation	6	37.7×	3e-06
establishment or maintenance of epithelial cell apical/basal polarity	9	37.1×	2e-09
receptor clustering	7	31.0×	1e-06
regulation of postsynaptic membrane neurotransmitter receptor levels	6	21.1×	4e-05
platelet-derived growth factor receptor signaling pathway	5	19.9×	3e-04
protein dephosphorylation	11	17.3×	3e-08
positive regulation of blood vessel endothelial cell migration	6	16.7×	1e-04
T cell costimulation	6	15.9×	2e-04

Disease & clinical

Cancer significance

From intOGen — cancer-driver classification: activating (oncogene-like) across 3 cancer types — ANGS, NETNOS, UCS.

Clinical variants and AI predictions

ClinVar

848 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	20
Likely pathogenic	5
Uncertain significance	319
Likely benign	284
Benign	119

Top pathogenic / likely-pathogenic (25)

Variant ID	HGVS	Classification
2096300	NM_002609.4(PDGFRB):c.2483C>A (p.Ala828Glu)	Pathogenic
217122	NM_002609.4(PDGFRB):c.1751C>G (p.Pro584Arg)	Pathogenic
3233448	NM_002609.4(PDGFRB):c.2546_2548del (p.Arg849_Asp850delinsHis)	Pathogenic
3237207	NM_002609.4(PDGFRB):c.2548G>T (p.Asp850Tyr)	Pathogenic
3392579	NM_002609.4(PDGFRB):c.2476G>T (p.Asp826Tyr)	Pathogenic
3602096	N666Y	Pathogenic
375237	NM_002609.4(PDGFRB):c.1699A>G (p.Lys567Glu)	Pathogenic
375555	NM_002609.4(PDGFRB):c.1697_1702del (p.Trp566_Val568delinsLeu)	Pathogenic
375556	NM_002609.4(PDGFRB):c.1998C>G (p.Asn666Lys)	Pathogenic
375557	NM_002609.4(PDGFRB):c.1615_1616insGAT (p.Ile538_Leu539insArg)	Pathogenic
375681	NM_002609.4(PDGFRB):c.2549A>T (p.Asp850Val)	Pathogenic
375682	NM_002609.4(PDGFRB):c.1696T>C (p.Trp566Arg)	Pathogenic
39588	NM_002609.4(PDGFRB):c.1973T>C (p.Leu658Pro)	Pathogenic
495300	NM_002609.4(PDGFRB):c.1994T>C (p.Val665Ala)	Pathogenic
55848	NM_002609.4(PDGFRB):c.1681C>T (p.Arg561Cys)	Pathogenic
973194	NM_002609.4(PDGFRB):c.1519_1520insTACTGTCGGTGC (p.Val506_Arg507insLeuLeuSerVal)	Pathogenic
973195	NM_002609.4(PDGFRB):c.1716_1717insGAGCTGATCCGATGGAAGGTGATTGAGTCTGTG (p.Val572_Ser573insGluLeuIleArgTrpLysValIleGluSerVal)	Pathogenic
973196	NM_002609.4(PDGFRB):c.1682_1684del (p.Arg561_Tyr562delinsHis)	Pathogenic
973197	NM_002609.4(PDGFRB):c.1684T>G (p.Tyr562Asp)	Pathogenic
973198	NM_002609.4(PDGFRB):c.1613T>A (p.Ile538Asn)	Pathogenic
1333888	NM_002609.4(PDGFRB):c.1025C>T (p.Ala342Val)	Likely pathogenic
2432540	NM_002609.4(PDGFRB):c.2566A>T (p.Asn856Tyr)	Likely pathogenic
3393309	NM_002609.4(PDGFRB):c.1684T>A (p.Tyr562Asn)	Likely pathogenic
3773730	NM_002609.4(PDGFRB):c.2567A>T (p.Asn856Ile)	Likely pathogenic
4794434	NM_002609.4(PDGFRB):c.1A>G (p.Met1Val)	Likely pathogenic

SpliceAI

4470 predictions. Top by Δscore:

Variant	Effect	Δscore
5:150115944:GAGC:G	acceptor_loss	1.0000
5:150115945:AGCTA:A	acceptor_loss	1.0000
5:150115946:GCTA:G	acceptor_loss	1.0000
5:150115947:C:CC	acceptor_gain	1.0000
5:150117613:GTTAC:G	donor_loss	1.0000
5:150117614:TTACC:T	donor_loss	1.0000
5:150117615:TACCT:T	donor_loss	1.0000
5:150117616:A:T	donor_loss	1.0000
5:150117617:C:CG	donor_loss	1.0000
5:150117858:C:CT	acceptor_gain	1.0000
5:150117858:C:T	acceptor_gain	1.0000
5:150117859:G:T	acceptor_gain	1.0000
5:150117863:C:CT	acceptor_gain	1.0000
5:150117864:A:T	acceptor_gain	1.0000
5:150117873:C:CT	acceptor_gain	1.0000
5:150117873:C:T	acceptor_gain	1.0000
5:150117874:A:T	acceptor_gain	1.0000
5:150118743:ATAC:A	donor_loss	1.0000
5:150118744:TA:T	donor_loss	1.0000
5:150118745:A:AC	donor_gain	1.0000
5:150118746:C:CC	donor_gain	1.0000
5:150118746:C:G	donor_loss	1.0000
5:150118847:TCATA:T	acceptor_gain	1.0000
5:150118848:CATAG:C	acceptor_gain	1.0000
5:150118850:TAG:T	acceptor_gain	1.0000
5:150118850:TAGC:T	acceptor_loss	1.0000
5:150118851:AG:A	acceptor_gain	1.0000
5:150118852:GCTG:G	acceptor_loss	1.0000
5:150118853:C:CA	acceptor_loss	1.0000
5:150118853:C:CC	acceptor_gain	1.0000

AlphaMissense

7221 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
5:150118809:G:C	R948G	1.000
5:150118828:C:A	W941C	1.000
5:150118828:C:G	W941C	1.000
5:150118830:A:G	W941R	1.000
5:150118830:A:T	W941R	1.000
5:150118831:G:C	C940W	1.000
5:150118833:A:G	C940R	1.000
5:150119527:A:G	F913S	1.000
5:150119555:A:C	Y904D	1.000
5:150119557:G:T	P903H	1.000
5:150120026:T:A	E895V	1.000
5:150120026:T:G	E895A	1.000
5:150120027:C:T	E895K	1.000
5:150120030:A:G	W894R	1.000
5:150120030:A:T	W894R	1.000
5:150120032:A:G	L893P	1.000
5:150120035:A:G	L892P	1.000
5:150120041:C:T	G890E	1.000
5:150120042:C:A	G890W	1.000
5:150120042:C:G	G890R	1.000
5:150120042:C:T	G890R	1.000
5:150120047:G:A	S888F	1.000
5:150120049:C:A	W887C	1.000
5:150120049:C:G	W887C	1.000
5:150120051:A:G	W887R	1.000
5:150120051:A:T	W887R	1.000
5:150120056:T:A	D885V	1.000
5:150120056:T:C	D885G	1.000
5:150120056:T:G	D885A	1.000
5:150120057:C:A	D885Y	1.000

dbSNP variants (sampled 300 via entrez): RS1000076378 (5:150116492 C>A), RS1000105628 (5:150154477 A>G), RS1000124160 (5:150116814 C>T), RS1000216985 (5:150142667 T>A), RS1000260824 (5:150148542 A>G), RS1000315273 (5:150153954 G>T), RS1000340676 (5:150136927 C>T), RS1000413556 (5:150154740 G>A), RS1000427316 (5:150133041 C>G), RS1000763061 (5:150133249 G>A), RS1000900144 (5:150137591 G>A,C), RS1000904641 (5:150131728 G>A,C), RS1001020517 (5:150153345 C>T), RS1001044480 (5:150148170 C>A), RS1001064598 (5:150142768 A>C)

Disease associations

OMIM: gene MIM:173410 | disease phenotypes: MIM:601812, MIM:615007, MIM:616592, MIM:228550, MIM:131440, MIM:213600, MIM:606656, MIM:621091, MIM:220200, MIM:604169

GenCC curated gene-disease

Disease	Classification	Inheritance
skeletal overgrowth-craniofacial dysmorphism-hyperelastic skin-white matter lesions syndrome	Definitive	Autosomal dominant
acroosteolysis-keloid-like lesions-premature aging syndrome	Definitive	Autosomal dominant
basal ganglia calcification, idiopathic, 4	Strong	Autosomal dominant
myofibromatosis, infantile, 1	Strong	Autosomal dominant
bilateral striopallidodentate calcinosis	Supportive	Autosomal dominant
infantile myofibromatosis	Supportive	Autosomal dominant
ocular pterygium-digital keloid dysplasia syndrome	Limited	Autosomal dominant
hereditary progressive mucinous histiocytosis	Limited	Autosomal dominant

Mondo (17): acroosteolysis-keloid-like lesions-premature aging syndrome (MONDO:0011150), basal ganglia calcification, idiopathic, 4 (MONDO:0014004), skeletal overgrowth-craniofacial dysmorphism-hyperelastic skin-white matter lesions syndrome (MONDO:0014704), infantile myofibromatosis (MONDO:0016824), myofibromatosis, infantile, 1 (MONDO:0009227), myeloproliferative disorder, chronic, with eosinophilia (MONDO:0007546), basal ganglia calcification, idiopathic, 1 (MONDO:0024538), cerebral palsy (MONDO:0006497), intellectual disability (MONDO:0001071), ocular pterygium-digital keloid dysplasia syndrome (MONDO:0976136), hydrocephalus (MONDO:0001150), Dandy-Walker syndrome (MONDO:0009072), left ventricular noncompaction (MONDO:0018901), parkinsonian disorder (MONDO:0021095), microcephaly (MONDO:0001149)

Orphanet (7): Bilateral striopallidodentate calcinosis (Orphanet:1980), Infantile myofibromatosis (Orphanet:2591), Acroosteolysis-keloid-like lesions-premature aging syndrome (Orphanet:363665), Kosaki overgrowth syndrome (Orphanet:477831), Isolated Dandy-Walker malformation (Orphanet:217), Left ventricular noncompaction (Orphanet:54260), NON RARE IN EUROPE: Unexplained intellectual disability (Orphanet:319658)

HPO phenotypes

179 total (30 of 179 shown, HPO-id order):

HPO	Term
HP:0000002	Abnormality of body height
HP:0000006	Autosomal dominant inheritance
HP:0000012	Urinary urgency
HP:0000020	Urinary incontinence
HP:0000077	Abnormality of the kidney
HP:0000098	Tall stature
HP:0000169	Gingival fibromatosis
HP:0000219	Thin upper lip vermilion
HP:0000233	Thin vermilion border
HP:0000256	Macrocephaly
HP:0000270	Delayed cranial suture closure
HP:0000271	Abnormality of the face
HP:0000278	Retrognathia
HP:0000298	Mask-like facies
HP:0000307	Pointed chin
HP:0000316	Hypertelorism
HP:0000326	Abnormal maxilla morphology
HP:0000327	Hypoplasia of the maxilla
HP:0000336	Prominent supraorbital ridges
HP:0000347	Micrognathia
HP:0000407	Sensorineural hearing impairment
HP:0000422	Abnormal nasal bridge morphology
HP:0000426	Prominent nasal bridge
HP:0000431	Wide nasal bridge
HP:0000444	Convex nasal ridge
HP:0000460	Narrow nose
HP:0000478	Abnormality of the eye
HP:0000494	Downslanted palpebral fissures
HP:0000508	Ptosis
HP:0000520	Proptosis

GWAS associations

8 associations (top):

Study	Trait	p-value
GCST004608_18	Granulocyte percentage of myeloid white cells	7.000000e-36
GCST004609_57	Monocyte percentage of white cells	2.000000e-46
GCST004625_17	Monocyte count	1.000000e-54
GCST006585_487	Blood protein levels	0.000000e+00
GCST009244_7	Cytokine network levels (multivariate analysis)	4.000000e-09
GCST90000584_8	Inflammatory biomarkers (multivariate analysis)	1.000000e-15
GCST90002393_107	Monocyte count	1.000000e-129
GCST90002394_31	Monocyte percentage of white cells	1.000000e-128

EFO canonical traits (11, from GWAS)

EFO ID	Trait name
EFO:0007997	granulocyte percentage of myeloid white cells
EFO:0007989	monocyte percentage of leukocytes
EFO:0005091	monocyte count
EFO:0004750	interleukin 10 measurement
EFO:0004753	interleukin 12 measurement
EFO:0004810	interleukin-6 measurement
EFO:0008165	interferon gamma measurement
EFO:0008174	interleukin 17 measurement
EFO:0008184	interleukin 4 measurement
EFO:0008293	stromal cell-derived factor 1 alpha measurement
EFO:0004872	inflammatory biomarker measurement

MeSH disease descriptors (13)

Descriptor	Name	Tree numbers
D002547	Cerebral Palsy	C10.228.140.140.254
D003616	Dandy-Walker Syndrome	C10.228.140.252.300; C10.228.140.602.500; C10.500.205; C16.131.666.205
D006849	Hydrocephalus	C10.228.140.602
D008607	Intellectual Disability	C10.597.606.360; C23.888.592.604.646; F01.700.687; F03.625.539
D008831	Microcephaly	C05.660.207.620; C10.500.507.400.500; C16.131.621.207.620; C16.131.666.507.400.500
D018224	Myofibromatosis	C04.557.450.565.590.550
D020734	Parkinsonian Disorders	C10.228.140.079.862; C10.228.662.600
C537657	Basal ganglia calcification, idiopathic 2 (supp.)
C536275	Fahr’s disease (supp.)
C562978	Fibromatosis, Congenital Generalized (supp.)
C564186	Histiocytosis, Progressive Mucinous (supp.)
C565054	Myeloproliferative Disorder, Chronic, with Eosinophilia (supp.)
C536653	Penttinen-Aula syndrome (supp.)

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (5): CHEMBL1913 (SINGLE PROTEIN), CHEMBL2095189 (PROTEIN COMPLEX), CHEMBL2111440 (SELECTIVITY GROUP), CHEMBL4523729 (PROTEIN-PROTEIN INTERACTION), CHEMBL4879531 (PROTEIN-PROTEIN INTERACTION)

Molecules with ChEMBL bioactivity

102 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 348,070 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

Molecule	Name	Phase	Patents
CHEMBL1171837	PONATINIB	4	8,955
CHEMBL1287853	FEDRATINIB	4	3,554
CHEMBL1289494	TIVOZANIB	4	4,455
CHEMBL1289601	LENVATINIB	4	8,784
CHEMBL1289926	AXITINIB	4	15,732
CHEMBL1336	SORAFENIB	4	86,060
CHEMBL1567	SUNITINIB MALATE	4	161
CHEMBL1946170	REGORAFENIB	4	12,678
CHEMBL2035187	PACRITINIB	4	3,345
CHEMBL24828	VANDETANIB	4	42,230
CHEMBL255863	NILOTINIB	4	38,627
CHEMBL288441	BOSUTINIB	4	12,255
CHEMBL3039504	NINTEDANIB ESYLATE	4	659
CHEMBL3301622	GILTERITINIB	4	2,395
CHEMBL3348923	TOVORAFENIB	4	834
CHEMBL3813873	PEXIDARTINIB	4	3,586
CHEMBL477772	PAZOPANIB	4	15,540
CHEMBL502835	NINTEDANIB	4	8,545
CHEMBL535	SUNITINIB	4	79,020
CHEMBL5416410	DASATINIB	4	655
CHEMBL553	ERLOTINIB	4
CHEMBL554	LAPATINIB	4
CHEMBL576982	QUIZARTINIB	4
CHEMBL608533	MIDOSTAURIN	4
CHEMBL941	IMATINIB	4
CHEMBL101253	VATALANIB	3
CHEMBL1278146	FAMITINIB	3
CHEMBL1908391	MASITINIB	3
CHEMBL2105728	CRENOLANIB	3
CHEMBL217092	SARACATINIB	3

Clinical evidence (CIViC)

Drug × variant × indication: 2 predictive associations from 2 curated evidence items.

Variant	Therapy	Indication	Effect	Level	CIViC
PDGFRB Overexpression	Sunitinib	Dermatofibrosarcoma Protuberans	Sensitivity/Response	CIViC C	EID7992
AGGF1::PDGFRB Fusion AND PDGFRB C843G	CHZ868	Childhood B-cell Acute Lymphoblastic Leukemia	Sensitivity/Response	CIViC D	EID6999

PharmGKB: 1 entry (VIP=true, CPIC=false)

PharmGKB variants

4 variants.

Variant	Genes	Level	Score	#Clin annots	Drugs
rs2302273	PDGFRB		0.00	0
rs797044887	PDGFRB		0.00	0
rs201866603	PDGFRB		0.00	0
rs55712339	CSF1R, PDGFRB		0.00	0

GtoPdb / IUPHAR curated pharmacology

(IUPHAR/BPS Guide to Pharmacology — expert-curated)

Target class: catalytic receptor — Type III RTKs: PDGFR, CSFR, Kit, FLT3 receptor family

Most potent curated ligand interactions (48 total), top 25:

Ligand	Action	Affinity	Parameter
CP-673451	Inhibition	9.0	pIC50
AC710	Inhibition	9.0	pKd
compound 8h [PMID: 22765894]	Inhibition	8.89	pIC50
compound 8i [PMID: 22765894]	Inhibition	8.82	pIC50
velzatinib	Inhibition	8.59	pIC50
SU11652	Inhibition	8.52	pIC50
crenolanib	Inhibition	8.49	pKd
SU-14813	Inhibition	8.4	pIC50
famitinib	Inhibition	8.4	pIC50
PDGF receptor tyrosine kinase inhibitor IV	Inhibition	8.38	pIC50
cediranib	Inhibition	8.3	pIC50
dovitinib	Inhibition	8.3	pIC50
quizartinib	Inhibition	8.11	pKd
sunitinib	Inhibition	8.1	pKi
lucitanib	Inhibition	8.1	pIC50
henatinib	Inhibition	7.96	pIC50
PDGF RTK inhibitor	Inhibition	7.92	pIC50
ilorasertib	Inhibition	7.89	pIC50
naporafenib	Inhibition	7.85	pKd
tafetinib	Inhibition	7.85	pIC50
MK-2461	Inhibition	7.66	pIC50
KBP-7018	Inhibition	7.6	pIC50
compound 5e [PMID: 28580438]	Inhibition	7.55	pKd
BPR1R024	Inhibition	7.5	pIC50
orantinib	Inhibition	7.3	pIC50

Binding affinities (BindingDB)

928 measured of 1161 human assays (1179 total across all organisms); most potent 50 below. Values come from heterogeneous assays and are not directly comparable.

Ligand	Measure	Value	Patent
1-Phenylbenzimidazole deriv. 76	EC50	0.00399 nM
N-(5-((2-(7- azabicyclo[2.2.1]heptan-7- yl)ethyl)carbamoyl)-2- methylpyridin-3-yl)-2-(1,5- dimethyl-1H-pyrazol-4- yl)pyrazolo[5,1-b]thiazole-7- carboxamide	IC50	0.3 nM	US-20240109917: PYRAZOLOTHIAZOLE CARBOXAMIDES AND THEIR USES AS PDGFR INHIBITORS
1-[2-(4-methylphenyl)-5-tert-butyl-pyrazol-3-yl]-3-[4-(2-morpholin-4-ylethoxy)naphthalen-1-yl]urea	KD	0.37 nM
5-{[(5Z)-4-[(3-chloro-4-fluorophenyl)amino]-6-oxo-5H,6H,7H-pyrrolo[2,3-d]pyrimidin-5-ylidene]methyl}-N-[3-(diethylamino)-2-hydroxypropyl]-4-methyl-1H-pyrrole-2-carboxamide	IC50	0.45 nM
8-[4-amino-3-[4-[(2-fluoro-5-methylphenyl)carbamoylamino]phenyl]pyrazolo[3,4-d]pyrimidin-1-yl]-N-hydroxyoctanamide	IC50	0.47 nM	US-9695181: Hydroximic acid derivatives and medical applications therof
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-({[2-(pyridin-4-yl)ethyl]amino}methyl)-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.475 nM	US-10047096
8-{[Ethyl(methyl)amino]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.487 nM	US-10047096
1-[4-(cyanomethyl)-1-(1H-indol-7-ylmethyl)piperidin-4-yl]-3-(4-fluoroanilino)pyrazole-4-carboxamide	IC50	0.626 nM	US-10047096
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-{[(2S,4R)-4-hydroxy-2-(hydroxymethyl)pyrrolidin-1-yl]methyl}-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.68 nM	US-10047096
8-{[(2-Aminoethyl)(ethyl)amino]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.727 nM	US-10047096
US20250360145, Example 119	IC50	0.73 nM	US-20250360145: CSF-1R INHIBITORS AND USES THEREOF
8-{[(2-Aminoethyl)(methyl)amino]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.739 nM	US-10047096
4-[(4-Chloro-2-fluoro-5-hydroxyphenyl)amino]-8-{[ethyl(2-hydroxyethyl)amino]methyl}-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.793 nM	US-10047096
tert-butyl 4-[4-carbamoyl-3-(pyridazin-3-ylamino)pyrazol-1-yl]-4-(cyanomethyl)piperidine-1-carboxylate	IC50	0.826 nM	US-10047096
8-{[(3R)-3-Aminopiperidin-1-yl]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.829 nM	US-10047096
US20250360145, Example 87	IC50	0.83 nM	US-20250360145: CSF-1R INHIBITORS AND USES THEREOF
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-(piperazin-1-ylmethyl)-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.861 nM	US-10047096
8-{[Ethyl(2-methoxyethyl)amino]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.905 nM	US-10047096
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-[(4-hydroxypiperidin-1-yl)methyl]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.928 nM	US-10047096
8-[(Diethylamino)methyl]-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.953 nM	US-10047096
8-{[Butyl(ethyl)amino]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.962 nM	US-10047096
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-{[(2-hydroxyethyl)(methyl)amino]methyl}-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.962 nM	US-10047096
8-{[(1,3-Dihydroxypropan-2-yl)amino]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.974 nM	US-10047096
tert-butyl 4-[4-carbamoyl-3-[(1-oxo-2,3-dihydroisoindol-5-yl)amino]pyrazol-1-yl]-4-(cyanomethyl)piperidine-1-carboxylate	IC50	0.985 nM	US-10047096
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-{[(3S)-3-hydroxypyrrolidin-1-yl]methyl}-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	0.995 nM	US-10047096
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-[(isopropylamino)methyl]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.01 nM	US-10047096
8-{[Ethyl(3-hydroxypropyl)amino]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.01 nM	US-10047096
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-{[(3S)-3-hydroxypiperidin-1-yl]methyl}-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.01 nM	US-10047096
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-{[(3R)-3-hydroxypyrrolidin-1-yl]methyl}-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.03 nM	US-10047096
8-{[(3,4-cis-Dihydroxypyrrolidin-1-yl]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.03 nM	US-10047096
8-(Azepan-1-ylmethyl)-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.05 nM	US-10047096
tert-butyl 4-[4-carbamoyl-3-[4-(2-methoxy-2-oxoethyl)anilino]pyrazol-1-yl]-4-(cyanomethyl)piperidine-1-carboxylate	IC50	1.05 nM	US-10047096
1-[4-(cyanomethyl)-1-[(3-methoxycyclobutyl)methyl]piperidin-4-yl]-3-[(2-fluoro-4-pyridinyl)amino]pyrazole-4-carboxamide	IC50	1.05 nM	US-10047096
8-[(4-Aminopiperidin-1-yl)methyl]-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.06 nM	US-10047096
8-[4-amino-3-[4-[(3-methylphenyl)carbamoylamino]phenyl]pyrazolo[3,4-d]pyrimidin-1-yl]-N-hydroxyoctanamide	IC50	1.09 nM	US-9695181: Hydroximic acid derivatives and medical applications therof
8-[(Cyclopentylamino)methyl]-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one acetic acid salt	IC50	1.09 nM	US-10047096
8-{[Ethyl(2-hydroxyethyl)amino]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.1 nM	US-10047096
5-{[(5Z)-4-[(1-benzyl-1H-indol-5-yl)amino]-6-oxo-5H,6H,7H-pyrrolo[2,3-d]pyrimidin-5-ylidene]methyl}-4-methyl-N-[2-(morpholin-4-yl)ethyl]-1H-pyrrole-2-carboxamide	IC50	1.1 nM
8-{[(3R)-3-Aminopyrrolidin-1-yl]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.12 nM	US-10047096
8-{[(2-Ethoxyethyl) (ethyl)amino]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.13 nM	US-10047096
8-{[Ethyl(isopropyl)amino]methyl}-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.15 nM	US-10047096
BDBM227897	IC50	1.16 nM	US-10047096
1-[4-(cyanomethyl)-1-(pyrazolo[1,5-a]pyridin-3-ylmethyl)piperidin-4-yl]-3-[(2-fluoro-4-pyridinyl)amino]pyrazole-4-carboxamide	IC50	1.18 nM	US-10047096
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-[(4a-hydroxyoctahydroisoquinolin-2(1H)-yl)methyl]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.2 nM	US-10047096
[4-[[7-(2-chloro-5-hydroxyphenyl)-5-methyl-1,2,4-benzotriazin-3-yl]amino]phenyl]-piperazin-1-ylmethanone	IC50	1.2 nM	US-8481536: Benzotriazine inhibitors of kinases
5-{[(5Z)-4-[(3-chloro-4-fluorophenyl)amino]-6-oxo-5H,6H,7H-pyrrolo[2,3-d]pyrimidin-5-ylidene]methyl}-N-[2-(diethylamino)ethyl]-4-methyl-1H-pyrrole-2-carboxamide	IC50	1.2 nM
US20250360145, Example 83	IC50	1.2 nM	US-20250360145: CSF-1R INHIBITORS AND USES THEREOF
8-[(Ethylamino)methyl]-4-[(2-fluoro-5-hydroxy-4-methylphenyl)amino]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.22 nM	US-10047096
4-[(2-Fluoro-5-hydroxy-4-methylphenyl)amino]-8-{[(3-isopropoxycyclobutyl)amino]methyl}-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.22 nM	US-10047096
4-[(4-Chloro-2-fluoro-5-hydroxyphenyl)amino]-8-[(diethylamino)methyl]-6,11-dihydro-5H-pyrido[2,3-b][1,5]benzodiazepin-5-one	IC50	1.25 nM	US-10047096

ChEMBL bioactivities

2882 potent at pChembl≥5 of 3076 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
10.12	Kd	0.075	nM	SUNITINIB
10.05	Kd	0.089	nM	CHEMBL3752910
10.00	IC50	0.1	nM	CHEMBL526507
10.00	IC50	0.1	nM	CHEMBL495749
9.99	ED50	0.102	nM	CHEMBL3752910
9.90	Ki	0.1259	nM	CHEMBL196363
9.85	IC50	0.14	nM	CHEMBL5977738
9.70	IC50	0.2	nM	CHEMBL2023477
9.70	IC50	0.2	nM	CHEMBL2023485
9.70	Kd	0.2	nM	SUNITINIB
9.70	IC50	0.2	nM	CHEMBL495984
9.68	Kd	0.21	nM	SUNITINIB
9.59	IC50	0.26	nM	SORAFENIB
9.54	Kd	0.29	nM	SU-014813
9.54	Kd	0.29	nM	CHEMBL1908396
9.52	IC50	0.3	nM	CHEMBL2023476
9.52	IC50	0.3	nM	CHEMBL495762
9.52	IC50	0.3	nM	CHEMBL498551
9.52	IC50	0.3	nM	CHEMBL1940109
9.50	Ki	0.3162	nM	CHEMBL1999414
9.49	Kd	0.32	nM	CEDIRANIB
9.40	IC50	0.4	nM	CHEMBL2023482
9.40	IC50	0.4	nM	CHEMBL2347053
9.40	IC50	0.4	nM	CHEMBL498013
9.40	IC50	0.4	nM	CHEMBL1940273
9.40	IC50	0.4	nM	CHEMBL340284
9.40	IC50	0.4	nM	CHEMBL123928
9.40	Ki	0.3981	nM	CHEMBL1996649
9.38	IC50	0.42	nM	CHEMBL5881155
9.32	IC50	0.475	nM	CHEMBL5758096
9.31	IC50	0.487	nM	CHEMBL6052352
9.30	IC50	0.5	nM	CHEMBL2023486
9.30	IC50	0.5	nM	CHEMBL6028045
9.30	IC50	0.5	nM	CHEMBL13976
9.30	Ki	0.5012	nM	CHEMBL1965170
9.30	Ki	0.5012	nM	ILORASERTIB
9.24	Kd	0.57	nM	AXITINIB
9.22	IC50	0.6	nM	STAUROSPORINE
9.22	IC50	0.6	nM	CHEMBL495735
9.20	IC50	0.626	nM	CHEMBL5857889
9.20	Kd	0.63	nM	DASATINIB
9.20	Ki	0.631	nM	CHEMBL1988437
9.17	IC50	0.68	nM	CHEMBL5936239
9.15	IC50	0.7	nM	CHEMBL2023118
9.15	IC50	0.7	nM	CHEMBL495763
9.14	IC50	0.727	nM	CHEMBL5888997
9.13	IC50	0.739	nM	CHEMBL5990952
9.12	IC50	0.76	nM	STAUROSPORINE
9.10	IC50	0.793	nM	CHEMBL6007247
9.10	IC50	0.8	nM	CHEMBL1940108

PubChem BioAssay actives

2116 with measured affinity, of 5125 total; 50 most potent distinct compounds. Largely complementary to BindingDB; screening values are coarse (µM, 4 dp), so sub-nM hits tie at the floor.

Compound	Assay	Type	Value	Unit
4-amino-3-(1H-benzimidazol-2-yl)-5-(1-methylpiperidin-3-yl)oxy-1H-quinolin-2-one	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0001	uM
4-amino-N-benzyl-3-[6-(4-methylpiperazin-1-yl)-1H-benzimidazol-2-yl]-2-oxo-1H-quinoline-6-carboxamide	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0001	uM
4-methyl-3-[(1-methyl-6-pyridin-3-ylpyrazolo[3,4-d]pyrimidin-4-yl)amino]-N-[3-(trifluoromethyl)phenyl]benzamide	2148958: Binding affinity to human PDGFRB incubated for 45 mins by Kinobead based pull down assay	kd	0.0001	uM
Sunitinib	435926: Binding constant for PDGFRB kinase domain	kd	0.0001	uM
3-(1H-benzimidazol-2-yl)-4-[(4-ethyl-1-methylpiperidin-2-yl)amino]-1H-quinolin-2-one	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0002	uM
2-[4-(6,7-dimethoxyquinolin-4-yl)oxy-2-methoxyphenyl]-N-(4,5-dimethyl-1,2-oxazol-3-yl)acetamide	660685: Inhibition of PDGFRbeta	ic50	0.0002	uM
2-[4-(6,7-dimethoxyquinolin-4-yl)oxy-2-methoxyphenyl]-N-(5-ethyl-1H-pyrazol-3-yl)acetamide	660685: Inhibition of PDGFRbeta	ic50	0.0002	uM
5-[(Z)-(5-fluoro-2-oxo-1H-indol-3-ylidene)methyl]-N-[(2S)-2-hydroxy-3-morpholin-4-ylpropyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide	435926: Binding constant for PDGFRB kinase domain	kd	0.0003	uM
4-amino-3-(1H-benzimidazol-2-yl)-5-(4-methylpiperazin-1-yl)-1H-quinolin-2-one	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0003	uM
4-amino-5-chloro-3-[6-(4-methylpiperazin-1-yl)-1H-benzimidazol-2-yl]-1H-quinolin-2-one	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0003	uM
2-[4-[6-methoxy-7-(2-methoxyethoxy)quinazolin-4-yl]oxypyrazol-1-yl]-N-(3-methoxyphenyl)acetamide	639905: Inhibition of PDGFRbeta phosphorylation by cell based assay	ic50	0.0003	uM
2-[2-methoxy-4-(7-methoxyquinolin-4-yl)oxyphenyl]-N-(5-methyl-1,3-thiazol-2-yl)acetamide	660685: Inhibition of PDGFRbeta	ic50	0.0003	uM
1-[4-(6,7-dimethoxyquinolin-4-yl)oxy-2-methoxyphenyl]-3-[1-(1,3-thiazol-2-yl)ethyl]urea	624875: Binding constant for PDGFRB kinase domain	kd	0.0003	uM
4-[(4-fluoro-2-methyl-1H-indol-5-yl)oxy]-6-methoxy-7-(3-pyrrolidin-1-ylpropoxy)quinazoline	624875: Binding constant for PDGFRB kinase domain	kd	0.0003	uM
4-[6,7-bis(2-methoxyethoxy)quinazolin-4-yl]-N-(4-isoquinolin-5-yloxyphenyl)piperazine-1-carboxamide	161392: Inhibition of platelet-derived growth factor receptor beta phosphorylation in MG63 cells in the absence of plasma	ic50	0.0004	uM
4-[6,7-bis(2-methoxyethoxy)quinazolin-4-yl]-N-(4-naphthalen-2-yloxyphenyl)piperazine-1-carboxamide	161392: Inhibition of platelet-derived growth factor receptor beta phosphorylation in MG63 cells in the absence of plasma	ic50	0.0004	uM
4-amino-7-fluoro-3-[6-(4-methylpiperazin-1-yl)-1H-benzimidazol-2-yl]-1H-quinolin-2-one	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0004	uM
(3Z)-3-[[5-(2-nitrophenyl)-1H-pyrazol-4-yl]methylidene]-1H-indol-2-one	739577: Inhibition of PDGFRbeta (unknown origin) after 10 mins by mobility shift assay	ic50	0.0004	uM
2-[4-(6,7-dimethoxyquinolin-4-yl)oxyphenyl]-N-(1-ethylpyrazol-4-yl)acetamide	639905: Inhibition of PDGFRbeta phosphorylation by cell based assay	ic50	0.0004	uM
N-(5-ethyl-1,2-oxazol-3-yl)-2-[2-methoxy-4-(7-methoxyquinolin-4-yl)oxyphenyl]acetamide	660685: Inhibition of PDGFRbeta	ic50	0.0004	uM
N-[2-(dimethylamino)ethyl]-5-[(Z)-(5-fluoro-2-oxo-1H-indol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide	161263: Inhibition of Platelet-derived growth factor receptor beta (PDGF-Rbeta)	ic50	0.0005	uM
N-(5-ethyl-1H-pyrazol-3-yl)-2-[2-methoxy-4-(7-methoxyquinolin-4-yl)oxyphenyl]acetamide	660685: Inhibition of PDGFRbeta	ic50	0.0005	uM
N-(2-chloro-6-methylphenyl)-2-[[6-[4-(2-hydroxyethyl)piperazin-1-yl]-2-methylpyrimidin-4-yl]amino]-1,3-thiazole-5-carboxamide;hydrate	435926: Binding constant for PDGFRB kinase domain	kd	0.0006	uM
4-amino-6-(benzylamino)-3-[6-(4-methylpiperazin-1-yl)-1H-benzimidazol-2-yl]-1H-quinolin-2-one	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0006	uM
(2S,3R,4R,6R)-3-methoxy-2-methyl-4-(methylamino)-29-oxa-1,7,17-triazaoctacyclo[12.12.2.12,6.07,28.08,13.015,19.020,27.021,26]nonacosa-8,10,12,14,19,21,23,25,27-nonaen-16-one	1308973: Inhibition of PDGFRbeta (unknown origin) incubated for 1 hr by spectrophotometric analysis	ic50	0.0006	uM
Axitinib	624875: Binding constant for PDGFRB kinase domain	kd	0.0006	uM
4-amino-5-methyl-3-[6-(4-methylpiperazin-1-yl)-1H-benzimidazol-2-yl]-1H-quinolin-2-one	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0007	uM
N-(1-ethylpyrazol-4-yl)-2-[2-methoxy-4-(7-methoxyquinolin-4-yl)oxyphenyl]acetamide	660685: Inhibition of PDGFRbeta	ic50	0.0007	uM
trans-(1S,2S)-2-[[6-[(2-amino-3-chloro-4-pyridinyl)methoxy]-1,3-benzothiazol-2-yl]amino]cyclohexan-1-ol	1723265: Binding affinity to human wild type PDGFRbeta (V582 to Y1009 residues) expressed in bacterial expression system by kinome scan assay	kd	0.0008	uM
2-[4-(6,7-dimethoxyquinazolin-4-yl)oxypyrazol-1-yl]-N-(3-methoxyphenyl)acetamide	639905: Inhibition of PDGFRbeta phosphorylation by cell based assay	ic50	0.0008	uM
3-(1H-benzimidazol-2-yl)-4-(piperidin-4-ylmethylamino)-1H-quinolin-2-one	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0009	uM
4-amino-3-[6-(4-methylpiperazine-1-carbonyl)-1H-benzimidazol-2-yl]-1H-quinolin-2-one	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0009	uM
2-N-[4-(4-ethylpiperazin-1-yl)-3-methoxyphenyl]-4-N-(1H-indazol-6-yl)-5-methylpyrimidine-2,4-diamine	1903975: Inhibition of PDGFRbeta (unknown origin) by caliper mobility shift assay	ic50	0.0009	uM
2-[4-(6,7-dimethoxyquinazolin-4-yl)oxyphenyl]-N-(3-methoxyphenyl)acetamide	639905: Inhibition of PDGFRbeta phosphorylation by cell based assay	ic50	0.0009	uM
2-[4-(6,7-dimethoxyquinolin-4-yl)oxy-2-methoxyphenyl]-N-(1-ethylpyrazol-4-yl)acetamide	660685: Inhibition of PDGFRbeta	ic50	0.0009	uM
4-[6,7-bis(2-methoxyethoxy)quinazolin-4-yl]-N-[4-(1H-indol-4-yloxy)phenyl]piperazine-1-carboxamide	161392: Inhibition of platelet-derived growth factor receptor beta phosphorylation in MG63 cells in the absence of plasma	ic50	0.0010	uM
6,7-dimethoxy-N-[3-(trifluoromethyl)phenyl]-1,4-dihydroindeno[2,1-d]pyrazol-3-amine	256697: Antiproliferative activity against PDGF-BB stimulated HCASMC	ic50	0.0010	uM
1-(3-methyl-4-morpholin-4-ylphenyl)-3-[4-(3-methyl-2H-pyrazolo[3,4-b]pyridin-4-yl)phenyl]urea	1444973: Inhibition of human His-tagged PDGFRbeta (558 to 1106 residues) expressed in baculovirus by ELISA	ic50	0.0010	uM
4-amino-5-fluoro-3-[6-(4-methylpiperazin-1-yl)-1H-benzimidazol-2-yl]-1H-quinolin-2-one	1431275: Inhibition of recombinant PDGFRbeta (unknown origin) expressed in baculovirus infected insect cells using biotinylated peptide substrate GGLFDDPSYVNVQNL in presence of ATP incubated for 1 to 4 hrs by time resolved fluorescence assay	ic50	0.0010	uM
N-[2-(4-amino-2-oxo-1H-quinolin-3-yl)-3H-benzimidazol-5-yl]-2-(dimethylamino)-N-methylacetamide	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0010	uM
4-amino-6-fluoro-3-[6-(4-methylpiperazin-1-yl)-1H-benzimidazol-2-yl]-1H-quinolin-2-one	411861: Inhibition of human recombinant PDGFRbeta	ic50	0.0010	uM
4-chloro-2-[5-[[(1R)-2-hydroxy-1-phenylethyl]amino]-3-isoquinolin-6-yl-2-pyridinyl]phenol	1450719: Inhibition of recombinant PDGFR-beta (unknown origin) using poly (Glu,Tyr) 4:1 as substrate after 60 mins by ELISA	ic50	0.0010	uM
5-fluoro-2-[5-[[(1R)-2-hydroxy-1-phenylethyl]amino]-3-(3-methyl-2H-indazol-5-yl)-2-pyridinyl]phenol	1450719: Inhibition of recombinant PDGFR-beta (unknown origin) using poly (Glu,Tyr) 4:1 as substrate after 60 mins by ELISA	ic50	0.0010	uM
N-(3-tert-butylphenyl)-1-methyl-5-[[2-[5-(trifluoromethyl)-1H-imidazol-2-yl]-4-pyridinyl]oxy]benzimidazol-2-amine	1255119: Inhibition of PDGFRbeta (unknown origin)	ic50	0.0010	uM
N-(4-chlorophenyl)-6-(6,7-dimethoxyquinolin-4-yl)oxynaphthalene-1-carboxamide	326901: Inhibition of PDGFRbeta	ic50	0.0010	uM
N-(4,5-dimethyl-1,2-oxazol-3-yl)-2-[2-methoxy-4-(7-methoxyquinolin-4-yl)oxyphenyl]acetamide	660685: Inhibition of PDGFRbeta	ic50	0.0010	uM
1-N’-[3-fluoro-4-[6-methoxy-7-(3-morpholin-4-ylpropoxy)quinolin-4-yl]oxyphenyl]-1-N-(4-fluorophenyl)cyclopropane-1,1-dicarboxamide	624875: Binding constant for PDGFRB kinase domain	kd	0.0010	uM
Toceranib	161263: Inhibition of Platelet-derived growth factor receptor beta (PDGF-Rbeta)	ic50	0.0010	uM
5-[(Z)-(5-fluoro-2-oxo-1H-indol-3-ylidene)methyl]-2,4-dimethyl-N-(pyridin-4-ylmethyl)-1H-pyrrole-3-carboxamide	161263: Inhibition of Platelet-derived growth factor receptor beta (PDGF-Rbeta)	ic50	0.0010	uM
N-[4-[(5-tert-butyl-1,2-oxazol-3-yl)carbamoylamino]phenyl]-5-(1-ethyl-2,2,6,6-tetramethylpiperidin-4-yl)oxypyridine-2-carboxamide	712313: Binding affinity to PDGFRbeta expressed in HEK-293 cells	kd	0.0010	uM

CTD chemical–gene interactions

126 total (human), top 30 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
(+)-JQ1 compound	decreases reaction, increases expression, decreases expression	5
Benzo(a)pyrene	decreases expression, decreases methylation, increases expression, increases methylation, affects methylation	5
Tetrachlorodibenzodioxin	affects expression, decreases expression	4
Sorafenib	affects cotreatment, decreases phosphorylation, decreases activity	3
lipoxin A4	affects reaction, increases phosphorylation, decreases reaction, increases activity, decreases phosphorylation (+1 more)	2
perfluorooctane sulfonic acid	affects expression, affects cotreatment, decreases expression	2
6,7-dimethoxy-2-phenylquinoxaline	increases expression, increases phosphorylation, decreases reaction	2
CP-673,451	decreases reaction, increases phosphorylation, decreases activity, decreases phosphorylation	2
ponatinib	decreases activity	2
Imatinib Mesylate	decreases expression, decreases phosphorylation	2
Dasatinib	increases expression, decreases phosphorylation	2
Resveratrol	decreases reaction, increases phosphorylation, affects binding	2
Aldrin	affects cotreatment, increases expression	2
Arsenic	affects expression, affects cotreatment, decreases expression, increases abundance	2
Copper	affects binding, decreases expression, increases expression	2
Dichlorodiphenyldichloroethane	affects cotreatment, increases expression	2
Dichlorodiphenyl Dichloroethylene	affects cotreatment, increases expression	2
Dieldrin	affects cotreatment, increases expression	2
Cyclosporine	decreases expression, increases expression	2
Copper Sulfate	decreases reaction, increases expression, decreases expression	2
Genistein	increases reaction, decreases expression, decreases reaction, increases activity, increases phosphorylation	2
Particulate Matter	decreases expression, increases abundance	2
GSK-J4	decreases expression	1
ammonium 2,3,3,3-tetrafluoro-2-(heptafluoropropoxy)-propanoate	affects expression, affects cotreatment	1
Baizhu	decreases expression	1
Fangfeng	decreases expression	1
dicrotophos	increases expression	1
methylmercuric chloride	increases expression	1
triphenyl phosphate	affects expression	1
bisphenol A	decreases expression	1

ChEMBL screening assays

1237 unique, capped per target: 1213 binding, 16 functional, 8 admet

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL1005541	Binding	Inhibition of Tel-fused PDGFRb kinase-mediated mouse BaF3 cell proliferation	Identification of NVP-TAE684, a potent, selective, and efficacious inhibitor of NPM-ALK. — Proc Natl Acad Sci U S A
CHEMBL1963778	Functional	PUBCHEM_BIOASSAY: Navigating the Kinome. (Class of assay: other) Panel member name: PDGFRB	PubChem BioAssay data set
CHEMBL4044107	ADMET	Inhibition of wild-type human partial length PDGFRB (V582 to Y1009 residues) expressed in Escherichia coli BL21 assessed as residual activity at 1000 nM after 30 mins by Kinomescan method relative to control	2-Oxo-3, 4-dihydropyrimido[4, 5-d]pyrimidinyl derivatives as new irreversible pan fibroblast growth factor receptor (FGFR) inhibitors. — Eur J Med Chem

Cellosaurus cell lines

27 cell lines: 18 spontaneously immortalized cell line, 9 cancer cell line

First 10 cell lines (id-ordered, not curated):

Cellosaurus	Name	Category	Sex
CVCL_1150	CTV-1	Cancer cell line	Male
CVCL_1326	Karpas-45	Cancer cell line	Male
CVCL_8150	MOLT-15	Cancer cell line	Male
CVCL_B0YI	Abcam SH-SY5Y PDGFRB KO	Cancer cell line	Female
CVCL_B8MA	Abcam HCT 116 PDGFRB KO	Cancer cell line	Male
CVCL_B9A5	Abcam MCF-7 PDGFRB KO	Cancer cell line	Female
CVCL_B9PH	Abcam A-549 PDGFRB KO	Cancer cell line	Male
CVCL_E0JT	Ubigene HeLa PDGFRB KO	Cancer cell line	Female
CVCL_E4X4	ZR-75-1 PDGFRA clone 30	Cancer cell line	Female
CVCL_XG30	TRMP PDGFRB-p.Arg634	Spontaneously immortalized cell line	Female

Clinical trials (associated diseases)

301 trials via MONDO — disease-level, not drug-specific.

Trial	Phase	Status	Title
NCT00154830	PHASE4	COMPLETED	Alterations of Functional Activities and Leg Stiffness After Hamstring Lengthening in Cerebral Palsy Children
NCT00432055	PHASE4	COMPLETED	Effects of Botulinum Toxin Type A in Adults With Cerebral Palsy
NCT00549471	PHASE4	TERMINATED	Improvement After Botulinum Toxin Injections to the Arms in Children With Cerebral Palsy
NCT00752934	PHASE4	TERMINATED	Does Oral Baclofen Improve Care and Comfort in Spastic Children in Nursing Homes?
NCT00964639	PHASE4	COMPLETED	Postoperative Pain in Children With Cerebral Palsy After Pelvic and Femoral Osteotomies
NCT01386255	PHASE4	WITHDRAWN	Placebo Controlled Study of Baclofen for GERD in Children With Cerebral Palsy
NCT02546999	PHASE4	COMPLETED	Does Botulinum Toxin A Make Walking Easier in Children With Cerebral Palsy?
NCT02633241	PHASE4	COMPLETED	A Pilot Study of Dexmedetomidine-Propofol in Children Undergoing Magnetic Resonance Imaging
NCT03117322	PHASE4	COMPLETED	Synbiotic, Prebiotics and Probiotics in Children With Cerebral Palsy and Constipation
NCT03648658	PHASE4	UNKNOWN	Paracetamol Study in Patients With Low Muscle Mass
NCT04074265	PHASE4	COMPLETED	Peri-operative Use of a Pain Injection in Pediatric Patients With Cerebral Palsy
NCT04273737	PHASE4	TERMINATED	Amantadine in Treating Cognitive & Motor Impairments in Adolescents and Adults With Cerebral Palsy
NCT04523935	PHASE4	COMPLETED	Excessive Crying in Children With Cerebral Palsy and Communication Deficits
NCT05887765	PHASE4	COMPLETED	Effect of Systematic Dexamethasone on the Duration of Popliteal Nerve Block for Anesthesia After Pediatric Ankle Surgery
NCT06176430	PHASE4	UNKNOWN	Comparison of Twice Weekly Versus Daily Iron Therapy in Treating Anemia in Children With Cerebral Palsy
NCT06189781	PHASE4	RECRUITING	Pain Injection Versus Epidural Anesthesia for Hip Surgery in Pediatric Patients With Cerebral Palsy
NCT00014989	PHASE3	COMPLETED	Beneficial Effects of Antenatal Magnesium Sulfate (BEAM Trial)
NCT00065949	PHASE3	UNKNOWN	Magnesium Sulfate to Prevent Brain Injury in Premature Infants
NCT00367068	PHASE3	COMPLETED	Dutch National ITB Study in Children With Cerebral Palsy
NCT00491894	PHASE3	COMPLETED	Safety and Efficacy Study of Oral Glycopyrrolate Liquid for the Treatment of Pathologic (Chronic Moderate to Severe) Drooling in Pediatric Patients 3 to 18 Years of Age With Cerebral Palsy or Other Neurologic Conditions
NCT00632528	PHASE3	COMPLETED	MEOPA to Improve Physical Therapy Results After Multilevel Surgery
NCT00822029	PHASE3	TERMINATED	Use of Oral Bisphosphonates in the Treatment of Osteoporosis of Non-walking Children With Cerebral Palsy
NCT00922077	PHASE3	COMPLETED	Individualized Neurodevelopmental Treatment
NCT01249417	PHASE3	COMPLETED	Dysport® Pediatric Lower Limb Spasticity Study
NCT01251380	PHASE3	COMPLETED	Dysport® Pediatric Lower Limb Spasticity Follow-on Study
NCT01437644	PHASE3	COMPLETED	The Post-Operative Pain in Cerebral Palsy (POPPIES) Trial
NCT01492608	PHASE3	COMPLETED	Magnesium Sulphate for Preterm Birth (MASP Study)
NCT01603602	PHASE3	COMPLETED	BOTOX® Treatment in Pediatric Upper Limb Spasticity
NCT01603615	PHASE3	COMPLETED	BOTOX® Open-Label Treatment in Pediatric Upper Limb Spasticity
NCT01603628	PHASE3	COMPLETED	BOTOX® Treatment in Pediatric Lower Limb Spasticity
NCT01603641	PHASE3	COMPLETED	BOTOX® Open-Label Treatment in Pediatric Lower Limb Spasticity
NCT01633736	PHASE3	UNKNOWN	Targeted Hip Strength Training in Children With Cerebral Palsy (CP)
NCT01898520	PHASE3	COMPLETED	A Safety, Efficacy and Tolerability Study of Sativex for the Treatment of Spasticity in Children Aged 8 to 18 Years
NCT01929434	PHASE3	COMPLETED	Efficacy of Stem Cell Transplantation Compared to Rehabilitation Treatment of Patients With Cerebral Paralysis
NCT02002884	PHASE3	COMPLETED	Dose-response Study of Efficacy and Safety of Botulinum Toxin Type A to Treat Spasticity of the Arm(s) or of Arm(s) and Leg(s) in Cerebral Palsy
NCT02270736	PHASE3	COMPLETED	Clinical Study to Investigate the Efficacy and Safety of NT 201 Compared to Placebo in the Treatment of Chronic Troublesome Drooling Associated With Neurological Disorders and/or Intellectual Disability
NCT02839785	PHASE3	TERMINATED	Analgesia and Physiotherapy in Children With Cerebral Palsy (ANTALKINECP)
NCT03110341	PHASE3	UNKNOWN	Effect of Erythropoietin in Premature Infants on White Matter Lesions and Neurodevelopmental Outcome
NCT03302871	PHASE3	COMPLETED	Integrated Management Enhances Functional Gains in Children With Cerebral Palsy Treated by BoNT-A
NCT03306212	PHASE3	COMPLETED	Efficacy of Intermittent Serial Casting on Spastic Wrist Flexion Deformity