FAT2

Summary

FAT2 (FAT atypical cadherin 2, HGNC:3596) is a protein-coding gene on chromosome 5q33.1, encoding Protocadherin Fat 2 (Q9NYQ8). Involved in the regulation of cell migration.

This gene is the second identified human homolog of the Drosophila fat gene, which encodes a tumor suppressor essential for controlling cell proliferation during Drosophila development. The gene product is a member of the cadherin superfamily, a group of integral membrane proteins characterized by the presence of cadherin-type repeats. In addition to containing 34 tandem cadherin-type repeats, the gene product has two epidermal growth factor (EGF)-like repeats and one laminin G domain. This protein most likely functions as a cell adhesion molecule, controlling cell proliferation and playing an important role in cerebellum development.

Source: NCBI Gene 2196 — RefSeq curated summary.

At a glance

• Gene–disease (curated): spinocerebellar ataxia 45 (Strong, GenCC)
• GWAS associations: 1
• Clinical variants (ClinVar): 1,476 total — 1 likely-pathogenic
• Phenotypes (HPO): 8
• Cancer driver (intOGen): loss-of-function (tumor-suppressor-like) across 1 cancer types
• MANE Select transcript: NM_001447

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:3596
Approved symbol	FAT2
Name	FAT atypical cadherin 2
Location	5q33.1
Locus type	gene with protein product
Status	Approved
Aliases	MEGF1, CDHF8, HFAT2, CDHR9
Ensembl gene	ENSG00000086570
Ensembl biotype	protein_coding
OMIM	604269
Entrez	2196

Gene structure

Transcript identifiers

Ensembl transcripts: 2 — 2 protein_coding

ENST00000261800, ENST00000520200

RefSeq mRNA: 1 — MANE Select: NM_001447 NM_001447

CCDS: CCDS4317

Canonical transcript exons

ENST00000261800 — 24 exons

Exon	Start	End
ENSE00000767790	151507154	151507611
ENSE00000767791	151510021	151510174
ENSE00000767792	151512165	151512606
ENSE00000906375	151534409	151534642
ENSE00001033801	151540567	151540763
ENSE00001085813	151529178	151529392
ENSE00001085814	151521276	151522086
ENSE00001085815	151525768	151525965
ENSE00001085818	151527996	151528133
ENSE00001085819	151527234	151527377
ENSE00001085820	151531587	151531970
ENSE00001085821	151517620	151517765
ENSE00001413330	151537793	151537946
ENSE00001820549	151504092	151506097
ENSE00002208176	151553177	151553387
ENSE00002220603	151551467	151551606
ENSE00002226428	151554362	151554673
ENSE00002239592	151563325	151563639
ENSE00002262397	151549295	151549505
ENSE00002265077	151556344	151556402
ENSE00002273025	151550590	151550871
ENSE00002287987	151565673	151568951
ENSE00002305057	151542285	151546337
ENSE00003917608	151591165	151591331

Expression profiles

Bgee: expression breadth ubiquitous, 182 present calls, max score 98.34.

FANTOM5 (CAGE): breadth broad, TPM avg 4.5026 / max 815.3420, expressed in 213 samples.

FANTOM5 promoters (4 alternative TSS)

Promoter ID	TPM avg	Samples expressed
64373	4.2841	210
64372	0.1260	59
64374	0.0774	12
64366	0.0152	5

Top tissues by expression

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

Tissue	Anatomy ID	Expression score	Quality
paraflocculus	UBERON:0005351	98.34	gold quality
cerebellar vermis	UBERON:0004720	97.25	gold quality
cerebellar cortex	UBERON:0002129	97.13	gold quality
cerebellum	UBERON:0002037	97.07	gold quality
cerebellar hemisphere	UBERON:0002245	97.07	gold quality
right hemisphere of cerebellum	UBERON:0014890	95.74	gold quality
lower esophagus mucosa	UBERON:0035834	91.50	gold quality
esophagus mucosa	UBERON:0002469	91.39	gold quality
gingival epithelium	UBERON:0001949	90.93	gold quality
gingiva	UBERON:0001828	90.59	gold quality
skin of abdomen	UBERON:0001416	89.06	gold quality
skin of leg	UBERON:0001511	88.10	gold quality
upper arm skin	UBERON:0004263	87.94	gold quality
zone of skin	UBERON:0000014	87.48	gold quality
epithelium of esophagus	UBERON:0001976	86.75	gold quality
esophagus squamous epithelium	UBERON:0006920	86.68	gold quality
oral cavity	UBERON:0000167	85.39	gold quality
tongue squamous epithelium	UBERON:0006919	85.11	silver quality
squamous epithelium	UBERON:0006914	84.60	gold quality
upper leg skin	UBERON:0004262	84.22	gold quality
mammalian vulva	UBERON:0000997	83.79	gold quality
olfactory segment of nasal mucosa	UBERON:0005386	83.79	gold quality
skin of hip	UBERON:0001554	82.59	gold quality
penis	UBERON:0000989	81.21	gold quality
secondary oocyte	CL:0000655	80.48	gold quality
nasal cavity epithelium	UBERON:0005384	79.92	silver quality
vagina	UBERON:0000996	79.27	gold quality
cervix epithelium	UBERON:0004801	78.84	silver quality
pharyngeal mucosa	UBERON:0000355	76.48	gold quality
tonsil	UBERON:0002372	76.19	gold quality

Single-cell (SCXA)

Detected in 1 experiment(s), a significant marker in 1.

Experiment	Marker?	Max mean expression
E-ANND-3	yes	10.09

Regulation

Is transcription factor: no

miRNA regulators (miRDB)

83 targeting FAT2, 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-8485	100.00	77.57	4731
HSA-MIR-3662	99.99	73.82	5684
HSA-MIR-3667-3P	99.99	67.17	1636
HSA-MIR-1236-3P	99.94	68.04	1695
HSA-MIR-6721-5P	99.93	68.92	2981
HSA-MIR-106A-5P	99.90	73.94	2683
HSA-MIR-17-5P	99.89	73.83	2665
HSA-MIR-6783-3P	99.89	67.92	2059
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-221-3P	99.86	71.56	1329
HSA-MIR-222-3P	99.86	71.35	1337
HSA-MIR-4728-5P	99.85	69.39	4718
HSA-MIR-4447	99.85	67.81	2900
HSA-MIR-548AZ-5P	99.83	69.94	3230
HSA-MIR-548T-5P	99.83	69.91	3220
HSA-MIR-6785-5P	99.82	68.68	4428
HSA-MIR-6756-5P	99.82	67.97	2466
HSA-MIR-149-3P	99.72	68.22	3963
HSA-MIR-6883-5P	99.69	68.05	3785
HSA-MIR-6766-5P	99.68	67.70	2325
HSA-MIR-6892-3P	99.68	66.40	1178
HSA-MIR-545-5P	99.66	70.18	2308
HSA-MIR-10393-5P	99.65	68.01	1368
HSA-MIR-4472	99.56	66.08	1478
HSA-MIR-302B-5P	99.50	69.49	1857

Literature-anchored findings (GeneRIF, showing 4)

• Human Fat2 is localized at immature adherens junctions in epidermal keratinocytes. (PMID:17900869)
• DeltaNp63alpha (TP63) is co-expressed with FAT2 and Slug in patient tumors and the elevated expression of DeltaNp63alpha, FAT2 and Slug correlated with poor patient outcome. (PMID:27081041)
• Requirement of FAT and DCHS protocadherins during hypothalamic-pituitary development. (PMID:33108146)
• Identification of the RP11-21C4.1/SVEP1 gene pair associated with FAT2 mutations as a potential biomarker in gastric cancer. (PMID:34308747)

Cross-species orthologs

7 orthologs

Organism	Symbol	Gene ID
danio_rerio	fat2	ENSDARG00000018923
mus_musculus	Fat2	ENSMUSG00000055333
rattus_norvegicus	Fat2	ENSRNOG00000012575
drosophila_melanogaster	stan	FBGN0024836
caenorhabditis_elegans		WBGENE00001475
caenorhabditis_elegans	hmr-1	WBGENE00001980
caenorhabditis_elegans	Y52B11A.11	WBGENE00014914

Paralogs (6): CELSR3 (ENSG00000008300), CELSR1 (ENSG00000075275), FAT1 (ENSG00000083857), CELSR2 (ENSG00000143126), FAT3 (ENSG00000165323), FAT4 (ENSG00000196159)

Protein

Protein identifiers

Protocadherin Fat 2 — Q9NYQ8 (reviewed: Q9NYQ8)

Alternative names: Cadherin family member 8, Multiple epidermal growth factor-like domains protein 1

All UniProt accessions (2): Q9NYQ8, H0YBK2

UniProt curated annotations — full annotation on UniProt →

Function. Involved in the regulation of cell migration. May be involved in mediating the organization of the parallel fibers of granule cells during cerebellar development.

Subunit / interactions. Homodimer.

Subcellular location. Cell membrane. Cell junction. Golgi apparatus. trans-Golgi network.

Tissue specificity. Expressed in epidermal keratinocytes, infant brain, cerebellum, and also in a variety of tumors, such as pancreatic cancer, diffuse type gastric cancer, ovarian cancer, esophageal cancer, skin squamous cell carcinoma, head and neck cancer. Not expressed in melanoma cell line A375 cells, normal epidermal melanocytes or normal dermal fibroblasts. Expressed in epidermal keratinocytes and squamous cell carcinoma (at protein level).

Disease relevance. Spinocerebellar ataxia 45 (SCA45) [MIM:617769] A form of spinocerebellar ataxia, a clinically and genetically heterogeneous group of cerebellar disorders. Patients show progressive incoordination of gait and often poor coordination of hands, speech and eye movements, due to degeneration of the cerebellum with variable involvement of the brainstem and spinal cord. SCA45 is a slowly progressive, autosomal dominant form with onset in adulthood. The disease may be caused by variants affecting the gene represented in this entry.

RefSeq proteins (1): NP_001438* (*=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR000742	EGF	Domain
IPR001791	Laminin_G	Domain
IPR001881	EGF-like_Ca-bd_dom	Domain
IPR002126	Cadherin-like_dom	Domain
IPR013320	ConA-like_dom_sf	Homologous_superfamily
IPR015919	Cadherin-like_sf	Homologous_superfamily
IPR020894	Cadherin_CS	Conserved_site
IPR039808	Cadherin	Family

Pfam: PF00008, PF00028, PF02210

UniProt features (113 total): glycosylation site 38, domain 35, sequence variant 25, disulfide bond 7, sequence conflict 3, topological domain 2, signal peptide 1, chain 1, transmembrane region 1

Structure

Experimental structures (PDB)

0 structures.

Predicted structure (AlphaFold)

No AlphaFold model available for Q9NYQ8 — AlphaFold DB does not currently provide models for proteins above ~3000 aa.

Functional residue map

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

Disulfide bonds (7): 3912–3944, 3951–3962, 3956–3972, 3974–3983, 3990–4001, 3995–4010, 4012–4021

Glycosylation sites (38): 39, 210, 280, 330, 459, 568, 627, 655, 789, 996, 1175, 1383, 1417, 1904, 1998, 2007, 2165, 2183, 2325, 2368 …

Function

Pathways and Gene Ontology

Reactome pathways

0 pathways

MSigDB gene sets: 123 (showing top): WU_APOPTOSIS_BY_CDKN1A_VIA_TP53, JAEGER_METASTASIS_DN, GOBP_AMEBOIDAL_TYPE_CELL_MIGRATION, AGGCACT_MIR5153P, MODULE_66, GOBP_CELL_CELL_ADHESION, RICKMAN_TUMOR_DIFFERENTIATED_WELL_VS_POORLY_DN, GOBP_TISSUE_MIGRATION, TGTGTGA_MIR377, MODULE_88, NAKAMURA_TUMOR_ZONE_PERIPHERAL_VS_CENTRAL_DN, GOCC_CELL_CELL_JUNCTION, GOBP_CELL_SUBSTRATE_ADHESION, MODULE_11, ACEVEDO_METHYLATED_IN_LIVER_CANCER_DN

GO Biological Process (6): homophilic cell-cell adhesion (GO:0007156), epithelial cell migration (GO:0010631), cell-substrate adhesion (GO:0031589), cell-cell adhesion mediated by cadherin (GO:0044331), cell adhesion (GO:0007155), cell-cell adhesion (GO:0098609)

GO Molecular Function (1): calcium ion binding (GO:0005509)

GO Cellular Component (7): Golgi apparatus (GO:0005794), plasma membrane (GO:0005886), adherens junction (GO:0005912), extracellular exosome (GO:0070062), cell-cell junction (GO:0005911), membrane (GO:0016020), anchoring junction (GO:0070161)

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
cell-cell adhesion	2
cell adhesion	2
ameboidal-type cell migration	1
epithelium migration	1
cellular process	1
metal ion binding	1
cytoplasm	1
endomembrane system	1
intracellular membrane-bounded organelle	1
membrane	1
cell periphery	1
cell-cell junction	1
extracellular vesicle	1
anchoring junction	1
cellular anatomical structure	1
cell junction	1

Protein interactions and networks

STRING

1212 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
FAT2	FJX1	Q86VR8	646
FAT2	LAMC1	P11047	603
FAT2	FAT4	Q6V0I7	582
FAT2	EGF	P01133	530
FAT2	SCRIB	Q14160	525
FAT2	ZNF750	Q32MQ0	524
FAT2	NPHS1	O60500	493
FAT2	CCDC175	P0C221	483
FAT2	KIRREL3	Q8IZU9	472
FAT2	KIRREL1	Q96J84	461
FAT2	PCDH11X	Q9BZA7	456
FAT2	GRM4	Q14833	453
FAT2	DDN	O94850	444
FAT2	GABRA6	Q16445	438
FAT2	FAT1	Q14517	424

IntAct

3 interactions, top by confidence:

A	B	Type	Score
PDLIM7	CRYBG2	psi-mi:“MI:0914”(association)	0.350
ATG16L1	ESYT2	psi-mi:“MI:0914”(association)	0.350

BioGRID (8): FAT2 (Affinity Capture-MS), FAT2 (Affinity Capture-MS), FAT2 (Reconstituted Complex), FAT2 (Proximity Label-MS), FAT2 (Cross-Linking-MS (XL-MS)), FAT2 (Affinity Capture-MS), FAT2 (Cross-Linking-MS (XL-MS)), FAT2 (Co-fractionation)

ESM2 similar proteins: E9Q7P9, O88277, O93319, P55280, P55281, P55285, P55289, P70408, P79995, P97326, Q08DJ5, Q12864, Q13634, Q14517, Q24298, Q2PZL6, Q3SWX5, Q5DWV1, Q5DWV2, Q5F226, Q63315, Q6B3P0, Q6KEQ9, Q6V0I7, Q6WXV7, Q6WYY1, Q6X862, Q6ZTQ4, Q71M42, Q8BIZ0, Q8BL00, Q8BM92, Q8BNA6, Q8N6Y1, Q8QGH3, Q8R508, Q8TDW7, Q90762, Q90763, Q91838

Diamond homologs: A2ASQ1, A2ASS6, A3KN33, A8DYP0, B4F785, O00468, O35474, O43854, O55005, O88516, O89026, O94779, P00740, P25304, P29294, P35590, Q05793, Q06561, Q16787, Q4LDE5, Q4VBE4, Q5R7K9, Q5RBN1, Q63HQ2, Q7TPD3, Q8N9C0, Q8TER0, Q8WZ42, Q95ND7, Q96MS0, Q9HCK4, Q9NYJ7, Q9NYQ8, Q9Y2H6, Q9Y6N7, Q9Z2I4, A2CG49, G5EBF1, O01761, O60229

SIGNOR signaling

0 interactions.

Disease & clinical

Cancer significance

From intOGen — cancer-driver classification: loss-of-function (tumor-suppressor-like) across 1 cancer types — VULVA.

Clinical variants and AI predictions

ClinVar

1476 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	0
Likely pathogenic	1
Uncertain significance	914
Likely benign	320
Benign	123

Top pathogenic / likely-pathogenic (1)

Variant ID	HGVS	Classification
3064881	NM_001447.3(FAT2):c.3632A>C (p.Glu1211Ala)	Likely pathogenic

SpliceAI

3493 predictions. Top by Δscore:

Variant	Effect	Δscore
5:151512189:T:TA	donor_gain	1.0000
5:151512190:C:A	donor_gain	1.0000
5:151523000:T:TA	donor_gain	1.0000
5:151525800:T:TA	donor_gain	1.0000
5:151525808:AGGC:A	donor_gain	1.0000
5:151525973:C:CT	acceptor_gain	1.0000
5:151525973:C:T	acceptor_gain	1.0000
5:151525974:G:T	acceptor_gain	1.0000
5:151527228:GCTTA:G	donor_loss	1.0000
5:151527229:CTTA:C	donor_loss	1.0000
5:151527230:TTAC:T	donor_loss	1.0000
5:151527231:TAC:T	donor_loss	1.0000
5:151527232:A:AC	donor_gain	1.0000
5:151527233:C:CC	donor_gain	1.0000
5:151527233:C:CT	donor_loss	1.0000
5:151527377:CCTAT:C	acceptor_gain	1.0000
5:151527378:C:CA	acceptor_loss	1.0000
5:151527378:C:CC	acceptor_gain	1.0000
5:151527379:T:G	acceptor_loss	1.0000
5:151527991:CTCA:C	donor_loss	1.0000
5:151527992:TCACC:T	donor_loss	1.0000
5:151527993:CAC:C	donor_loss	1.0000
5:151527994:A:AC	donor_gain	1.0000
5:151527995:C:A	donor_loss	1.0000
5:151527995:C:CC	donor_gain	1.0000
5:151528134:C:A	acceptor_loss	1.0000
5:151528135:T:A	acceptor_loss	1.0000
5:151529174:TTACC:T	donor_loss	1.0000
5:151529176:A:AC	donor_gain	1.0000
5:151529177:C:CC	donor_gain	1.0000

AlphaMissense

28601 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
5:151521583:G:C	S3670R	0.996
5:151521583:G:T	S3670R	0.996
5:151521585:T:G	S3670R	0.996
5:151543541:A:G	F2529S	0.996
5:151551504:A:T	V1420D	0.996
5:151528054:A:G	F3369S	0.995
5:151544393:G:T	A2245D	0.995
5:151553338:A:G	L1332P	0.995
5:151543493:C:G	R2545P	0.994
5:151544361:C:G	A2256P	0.994
5:151544394:C:G	A2245P	0.994
5:151544399:A:T	V2243D	0.994
5:151531607:A:G	F3264S	0.993
5:151542452:A:T	V2892D	0.993
5:151542680:A:T	V2816D	0.993
5:151543684:G:C	F2481L	0.993
5:151543684:G:T	F2481L	0.993
5:151543685:A:G	F2481S	0.993
5:151543686:A:G	F2481L	0.993
5:151554408:A:T	V1300D	0.993
5:151568687:A:G	F82S	0.993
5:151521641:A:G	L3651P	0.992
5:151542497:G:T	A2877D	0.992
5:151542654:C:G	D2825H	0.992
5:151543290:C:G	D2613H	0.992
5:151543301:G:T	A2609D	0.992
5:151544615:A:G	F2171S	0.992
5:151553327:A:G	W1336R	0.992
5:151553327:A:T	W1336R	0.992
5:151505937:G:C	F4226L	0.991

dbSNP variants (sampled 300 via entrez): RS1000074856 (5:151580737 G>A), RS1000141941 (5:151553873 G>C), RS1000190201 (5:151557066 C>G,T), RS1000241304 (5:151595982 C>T), RS1000272772 (5:151522487 T>C), RS1000284306 (5:151585649 A>G), RS1000331428 (5:151509020 CTCTT>C), RS1000424725 (5:151550400 G>A), RS1000425715 (5:151591129 C>A,G,T), RS1000429438 (5:151595776 G>C,T), RS1000431163 (5:151557553 A>G), RS1000510205 (5:151533466 G>C), RS1000518872 (5:151527672 C>G,T), RS1000552493 (5:151538163 G>A), RS1000597936 (5:151579836 T>C)

Disease associations

OMIM: gene MIM:604269 | disease phenotypes: MIM:108600, MIM:617769

GenCC curated gene-disease

Disease	Classification	Inheritance
spinocerebellar ataxia 45	Strong	Autosomal dominant

Mondo (3): spastic ataxia (MONDO:0017845), spinocerebellar ataxia 45 (MONDO:0033480), cerebellar ataxia (MONDO:0000437)

Orphanet (3): Spastic ataxia (Orphanet:316226), Spinocerebellar ataxia type 45 (Orphanet:589527), Rare ataxia (Orphanet:102002)

HPO phenotypes

8 total (8 of 8 shown, HPO-id order):

HPO	Term
HP:0000006	Autosomal dominant inheritance
HP:0001260	Dysarthria
HP:0001272	Cerebellar atrophy
HP:0002066	Gait ataxia
HP:0002070	Limb ataxia
HP:0003596	Middle age onset
HP:0003677	Slowly progressive
HP:0010545	Downbeat nystagmus

GWAS associations

1 associations (top):

Study	Trait	p-value
GCST011176_4	Stroke	6.000000e-07

MeSH disease descriptors (2)

Descriptor	Name	Tree numbers
D002524	Cerebellar Ataxia	C10.228.140.252.190; C10.597.350.090.500; C23.888.592.350.090.200
C564815	Spastic Ataxia (supp.)

Drugs & pharmacology

Drug and pharmacology data

Is drug target: no

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

CTD chemical–gene interactions

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

Chemical	Actions (top 5)	PubMed papers
sodium arsenite	decreases expression, increases abundance, increases expression	3
Benzo(a)pyrene	affects methylation, increases expression, increases methylation	3
Aflatoxin B1	affects methylation, increases expression	2
Particulate Matter	decreases expression, increases abundance	2
sotorasib	affects cotreatment, increases expression	1
bisphenol A	affects cotreatment, decreases methylation	1
cobaltous chloride	decreases expression	1
benzo(e)pyrene	decreases methylation	1
aflatoxin B2	decreases methylation	1
cupric chloride	decreases expression	1
S-(1,2-dichlorovinyl)cysteine	affects cotreatment, affects response to substance, increases expression	1
CGP 52608	affects binding, increases reaction	1
bisphenol S	decreases methylation	1
trametinib	affects cotreatment, increases expression	1
NVP-BKM120	affects cotreatment, increases expression	1
Resveratrol	affects cotreatment, decreases expression	1
Fulvestrant	affects cotreatment, decreases methylation	1
Air Pollutants	increases abundance, decreases expression	1
Arsenic	increases abundance, increases expression	1
Copper	affects cotreatment, decreases expression	1
Dichlorodiphenyl Dichloroethylene	increases expression	1
Lipopolysaccharides	increases expression, affects response to substance, affects cotreatment	1
Methapyrilene	decreases methylation	1
Nicotine	increases expression	1
Progesterone	decreases expression	1
Silicon Dioxide	decreases expression	1
Smoke	increases expression	1
Urethane	decreases expression	1
Sodium Selenite	decreases expression	1
Cadmium Chloride	decreases expression	1

Clinical trials (associated diseases)

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

Trial	Phase	Status	Title
NCT00950196	PHASE4	COMPLETED	Amantadine for Improving Neurologic Symptoms in Ataxia-Telangiectasia
NCT04107740	PHASE4	COMPLETED	C-Trelin Orally Disintegrated(OD) Tablet 5mg in Ataxia Due to Spinocerebellar Degeneration
NCT01970098	PHASE3	COMPLETED	A Confirmatory Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
NCT01970111	PHASE3	COMPLETED	An Extension Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
NCT01970124	PHASE3	COMPLETED	A Long-Term Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
NCT01970137	PHASE3	COMPLETED	A 24-week Open-label Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)
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NCT07040137	PHASE3	RECRUITING	Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration
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