GAREM2

gene
On this page

Also known as KIAA2038FLJ00375

Summary

GAREM2 (GRB2 associated regulator of MAPK1 subtype 2, HGNC:27172) is a protein-coding gene on chromosome 2p23.3, encoding GRB2-associated and regulator of MAPK protein 2 (Q75VX8). Probable adapter protein that may provide a link between cell surface epidermal growth factor receptor and the MAPK/ERK signaling pathway.

Predicted to act upstream of or within several processes, including cognition; neuron projection extension; and social behavior.

Source: NCBI Gene 150946 — RefSeq curated summary.

At a glance

  • Clinical variants (ClinVar): 426 total — 32 pathogenic, 43 likely-pathogenic
  • MANE Select transcript: NM_001168241

Identifiers

Gene identifiers

FieldValue
HGNC IDHGNC:27172
Approved symbolGAREM2
NameGRB2 associated regulator of MAPK1 subtype 2
Location2p23.3
Locus typegene with protein product
StatusApproved
AliasesKIAA2038, FLJ00375
Ensembl geneENSG00000157833
Ensembl biotypeprotein_coding
OMIM617999
Entrez150946

Gene structure

Transcript identifiers

Ensembl transcripts: 3 — 2 protein_coding, 1 protein_coding_CDS_not_defined

ENST00000401533, ENST00000407684, ENST00000496070

RefSeq mRNA: 2 — MANE Select: NM_001168241 NM_001168241, NM_001191033

CCDS: CCDS54336, CCDS54337

Canonical transcript exons

ENST00000401533 — 6 exons

ExonStartEnd
ENSE000007302652618618926186358
ENSE000010353372618423326185276
ENSE000010353412618296726183097
ENSE000014858562618723126189663
ENSE000014858652617634426176484
ENSE000014858692617308826173332

Expression profiles

Bgee: expression breadth ubiquitous, 187 present calls, max score 95.59.

FANTOM5 (CAGE): breadth ubiquitous, TPM avg 6.1852 / max 54.6453, expressed in 1333 samples.

FANTOM5 promoters (3 alternative TSS)

Promoter IDTPM avgSamples expressed
192512.2393503
192532.1823944
192521.7636826

Top tissues by expression

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

TissueAnatomy IDExpression scoreQuality
ventricular zoneUBERON:000305395.59gold quality
ganglionic eminenceUBERON:000402391.80gold quality
caudate nucleusUBERON:000187384.61gold quality
amygdalaUBERON:000187684.43gold quality
primordial germ cell in gonadCL:0000670 ∩ UBERON:000099184.06gold quality
putamenUBERON:000187483.61gold quality
hypothalamusUBERON:000189883.39gold quality
nucleus accumbensUBERON:000188283.21gold quality
medial globus pallidusUBERON:000247782.94gold quality
C1 segment of cervical spinal cordUBERON:000646982.48gold quality
secondary oocyteCL:000065582.39gold quality
anterior cingulate cortexUBERON:000983582.36gold quality
oocyteCL:000002382.10gold quality
right frontal lobeUBERON:000281082.03gold quality
substantia nigraUBERON:000203881.28gold quality
spinal cordUBERON:000224080.91gold quality
cortical plateUBERON:000534380.24gold quality
temporal lobeUBERON:000187180.12gold quality
globus pallidusUBERON:000187579.38gold quality
midbrainUBERON:000189179.28gold quality
neocortexUBERON:000195079.22gold quality
Brodmann (1909) area 9UBERON:001354078.80gold quality
Ammon’s hornUBERON:000195478.71gold quality
cerebral cortexUBERON:000095678.62gold quality
dorsolateral prefrontal cortexUBERON:000983478.58gold quality
forebrainUBERON:000189078.51gold quality
frontal cortexUBERON:000187078.29gold quality
prefrontal cortexUBERON:000045178.15gold quality
stromal cell of endometriumCL:000225577.54gold quality
brainUBERON:000095577.48gold quality

Single-cell (SCXA)

Detected in 1 experiment(s), a significant marker in 0.

ExperimentMarker?Max mean expression
E-ANND-3no2.54

Regulation

Is transcription factor: no

miRNA regulators (miRDB)

59 targeting GAREM2, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNAMax scoreAvg scoremiRNA target_count
HSA-MIR-1213699.9872.815713
HSA-MIR-548AA99.9670.643753
HSA-MIR-548AP-3P99.9670.643753
HSA-MIR-548T-3P99.9670.643753
HSA-MIR-1236-3P99.9468.041695
HSA-MIR-3121-3P99.8271.963630
HSA-MIR-57799.7869.132479
HSA-MIR-371499.7170.742671
HSA-MIR-117999.7168.701040
HSA-MIR-472999.6972.184233
HSA-MIR-4716-3P99.6966.731022
HSA-MIR-1212499.6869.172700
HSA-MIR-613499.6365.681537
HSA-MIR-182799.6368.573265
HSA-MIR-613299.6065.831554
HSA-MIR-6836-5P99.6065.621538
HSA-MIR-766-5P99.4767.912225
HSA-MIR-516A-3P99.4667.961378
HSA-MIR-516B-3P99.4667.961378
HSA-MIR-7162-5P99.4668.081368
HSA-MIR-431699.3765.751360
HSA-MIR-122B-3P99.2168.901333
HSA-MIR-429199.2068.882969
HSA-MIR-6815-3P99.1368.981530
HSA-MIR-465199.0667.572002
HSA-MIR-60898.9367.832013
HSA-MIR-2276-3P98.7667.751384
HSA-MIR-6501-3P98.7167.451480
HSA-MIR-6818-3P98.5668.231307
HSA-MIR-6776-5P98.5467.431304

Cross-species orthologs

3 orthologs

OrganismSymbolGene ID
danio_reriogaremlENSDARG00000073933
mus_musculusGarem2ENSMUSG00000044576
rattus_norvegicusGarem2ENSRNOG00000048004

Paralogs (1): GAREM1 (ENSG00000141441)

Protein

Protein identifiers

GRB2-associated and regulator of MAPK protein 2Q75VX8 (reviewed: Q75VX8)

Alternative names: GRB2-associated and regulator of MAPK1-like

All UniProt accessions (1): Q75VX8

UniProt curated annotations — full annotation on UniProt →

Function. Probable adapter protein that may provide a link between cell surface epidermal growth factor receptor and the MAPK/ERK signaling pathway.

Similarity. Belongs to the GAREM family.

Isoforms (3)

UniProt IDNamesCanonical?
Q75VX8-11yes
Q75VX8-22
Q75VX8-33

RefSeq proteins (2): NP_001161713, NP_001177962 (=MANE)

Domains & families (InterPro)

IDNameType
IPR013761SAM/pointed_sfHomologous_superfamily
IPR025946CABIT_domDomain
IPR052281GAREMFamily

Pfam: PF12736

UniProt features (23 total): compositionally biased region 6, region of interest 6, sequence conflict 5, splice variant 3, chain 1, domain 1, modified residue 1

Structure

Experimental structures (PDB)

0 structures.

Predicted structure (AlphaFold)

ModelpLDDTFraction very-high
AF-Q75VX8-F161.810.29

Functional residue map

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

Post-translational modifications (1): 735

Function

Pathways and Gene Ontology

Reactome pathways

0 pathways

MSigDB gene sets: 65 (showing top): GOBP_COGNITION, GOBP_BEHAVIOR, GOBP_NEURON_PROJECTION_EXTENSION, GOBP_GROWTH, GOBP_NEUROGENESIS, GOBP_BIOLOGICAL_PROCESS_INVOLVED_IN_INTRASPECIES_INTERACTION_BETWEEN_ORGANISMS, GOBP_DEVELOPMENTAL_CELL_GROWTH, GOBP_CELL_PROJECTION_ORGANIZATION, GOBP_DEVELOPMENTAL_GROWTH_INVOLVED_IN_MORPHOGENESIS, GOBP_CELL_GROWTH, GEORGES_TARGETS_OF_MIR192_AND_MIR215, GOBP_DEVELOPMENTAL_GROWTH, GOBERT_OLIGODENDROCYTE_DIFFERENTIATION_DN, PLASARI_TGFB1_TARGETS_10HR_UP, E2F5_TARGET_GENES

GO Biological Process (4): response to stress (GO:0006950), social behavior (GO:0035176), cognition (GO:0050890), neuron projection extension (GO:1990138)

GO Molecular Function (0):

GO Cellular Component (0):

GO top-level categories

Rollup of top GO terms by namespace:

CategoryTerms
response to stimulus1
behavior1
biological process involved in intraspecies interaction between organisms1
nervous system process1
developmental cell growth1
neuron projection morphogenesis1
developmental growth involved in morphogenesis1

Protein interactions and networks

STRING

632 interactions, top by confidence (×1000):

Protein AProtein BPartner UniProtScore
GAREM2FAM24AA6NFZ4511
GAREM2THEMIS2Q5TEJ8475
GAREM2ZNF562Q6V9R5434
GAREM2PM20D1Q6GTS8373
GAREM2MAB21L3Q8N8X9372
GAREM2KLHL35Q6PF15355
GAREM2FAM53AQ6NSI3349
GAREM2TTC23Q5W5X9348
GAREM2RNF182Q8N6D2324
GAREM2OR2L13Q8N349309
GAREM2CWC25Q9NXE8308
GAREM2PIGZQ86VD9304
GAREM2MVB12AQ96EY5303
GAREM2UMODL1Q5DID0300
GAREM2SPATA19Q7Z5L4296

IntAct

8 interactions, top by confidence:

ABTypeScore
GRB2WIPF3psi-mi:“MI:0914”(association)0.730
GRB2SH3PXD2Bpsi-mi:“MI:0914”(association)0.530
SYNGAP1POM121Cpsi-mi:“MI:0914”(association)0.350
GAREM1TNKSpsi-mi:“MI:0914”(association)0.350
GAREM1SCGB1D2psi-mi:“MI:0914”(association)0.350

BioGRID (27): GAREML (Affinity Capture-MS), GRB2 (Affinity Capture-Western), PTPN11 (Affinity Capture-Western), GAREML (Affinity Capture-MS), GAREML (Affinity Capture-MS), GAREML (Affinity Capture-MS), GAREML (Affinity Capture-MS), GAREML (Affinity Capture-RNA), GAREML (Affinity Capture-MS), GAREML (Proximity Label-MS), GAREML (Proximity Label-MS), GAREML (Proximity Label-MS), GAREML (Proximity Label-MS), GAREML (Proximity Label-MS), GAREML (Proximity Label-MS)

ESM2 similar proteins: A0A0U1RQ45, A0A1B0GWB2, A2A9T0, A6QPA0, A7MCY6, D3ZFB6, E9PUL5, E9Q0B3, F5GYI3, F5H4A9, J3QNX5, O70142, P0C1G7, P81408, P97764, P98077, Q148V8, Q15654, Q2KI80, Q3SX26, Q3SZL6, Q4V9L6, Q5FVJ4, Q5FW56, Q5RAC1, Q5T7N3, Q6DG50, Q6PAJ3, Q6PJ61, Q6ZMQ8, Q6ZNR0, Q6ZRV2, Q75VX8, Q7Z6L0, Q86UK7, Q86VE0, Q8BGW2, Q8BRJ3, Q8BX43, Q8C0R7

Diamond homologs: Q3UFT3, Q6NRE4, Q6PAJ3, Q75VX8, Q7ZVU1, Q9H706

SIGNOR signaling

0 interactions.

Disease & clinical

Clinical variants and AI predictions

ClinVar

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

ClassificationCount (floor)
Pathogenic32
Likely pathogenic43
Uncertain significance126
Likely benign149
Benign27

Top pathogenic / likely-pathogenic (30)

Variant IDHGVSClassification
1069908NM_000182.5(HADHA):c.1523del (p.Leu508fs)Pathogenic
1073572NM_000182.5(HADHA):c.1319dup (p.Ala441fs)Pathogenic
1075355NM_000182.5(HADHA):c.1528G>T (p.Glu510Ter)Pathogenic
1386792NM_000182.5(HADHA):c.1654dup (p.Ala552fs)Pathogenic
1390752NM_000182.5(HADHA):c.2200A>T (p.Lys734Ter)Pathogenic
1455936NM_000182.5(HADHA):c.1491_1495dup (p.Tyr499fs)Pathogenic
1459829NM_000182.5(HADHA):c.1540del (p.Thr514fs)Pathogenic
188712NM_000182.5(HADHA):c.1967del (p.Ile655_Leu656insTer)Pathogenic
194808NM_000182.5(HADHA):c.1918C>T (p.Gln640Ter)Pathogenic
194917NM_000182.5(HADHA):c.2146+1G>APathogenic
2020384NM_000182.5(HADHA):c.2010del (p.Asp670fs)Pathogenic
2025539NC_000002.12:g.26201318_26201321delPathogenic
2101164NM_000182.5(HADHA):c.2231del (p.Phe744fs)Pathogenic
2675968NM_000182.5(HADHA):c.1533dup (p.Ile512fs)Pathogenic
2921383NM_000182.5(HADHA):c.2011del (p.Glu671fs)Pathogenic
2922371NM_000182.5(HADHA):c.1361_1364dup (p.Lys455delinsAsnTer)Pathogenic
2934638NM_000182.5(HADHA):c.1231_1235del (p.Lys411fs)Pathogenic
2941242NM_000182.5(HADHA):c.2039_2040dup (p.Phe681fs)Pathogenic
2942406NM_000182.5(HADHA):c.1183dup (p.Thr395fs)Pathogenic
2943817NM_000182.5(HADHA):c.2063_2087dup (p.Glu699fs)Pathogenic
2947613NM_000182.5(HADHA):c.1249del (p.Ala416_Leu417insTer)Pathogenic
2950171NM_000182.5(HADHA):c.1921del (p.Glu641fs)Pathogenic
371597NM_000182.5(HADHA):c.1915_1918del (p.Tyr639fs)Pathogenic
3754541NM_000182.5(HADHA):c.1889del (p.Arg630fs)Pathogenic
4081703NM_000182.5(HADHA):c.1249_1259del (p.Ala416_Leu417insTer)Pathogenic
432660NM_000182.5(HADHA):c.1427_1428del (p.Thr476fs)Pathogenic
4784257NM_000182.5(HADHA):c.1586dup (p.Leu530fs)Pathogenic
801655NM_000182.5(HADHA):c.1689+2T>GPathogenic
848128NM_000182.5(HADHA):c.1690-17_1690-2delPathogenic
8729NM_000182.5(HADHA):c.1132C>T (p.Gln378Ter)Pathogenic

SpliceAI

1212 predictions. Top by Δscore:

VariantEffectΔscore
2:26173330:CAG:Cdonor_loss1.0000
2:26173331:AGG:Adonor_loss1.0000
2:26173332:GGT:Gdonor_loss1.0000
2:26173333:G:GAdonor_loss1.0000
2:26173334:T:Gdonor_loss1.0000
2:26176342:A:ACacceptor_loss1.0000
2:26176342:A:AGacceptor_gain1.0000
2:26176342:AG:Aacceptor_gain1.0000
2:26176342:AGG:Aacceptor_gain1.0000
2:26176343:G:GGacceptor_gain1.0000
2:26176343:GG:Gacceptor_gain1.0000
2:26176343:GGG:Gacceptor_gain1.0000
2:26176343:GGGGA:Gacceptor_gain1.0000
2:26176484:GGTG:Gdonor_loss1.0000
2:26176485:GT:Gdonor_loss1.0000
2:26176486:T:Adonor_loss1.0000
2:26182962:TGCA:Tacceptor_loss1.0000
2:26182964:CA:Cacceptor_loss1.0000
2:26182965:A:ATacceptor_loss1.0000
2:26182966:GGGAA:Gacceptor_gain1.0000
2:26183095:AAG:Adonor_loss1.0000
2:26183096:AG:Adonor_loss1.0000
2:26183097:GG:Gdonor_loss1.0000
2:26183099:T:Gdonor_loss1.0000
2:26188064:T:TAacceptor_gain1.0000
2:26176323:T:TAacceptor_gain0.9900
2:26176342:AGGG:Aacceptor_gain0.9900
2:26176343:GGGG:Gacceptor_gain0.9900
2:26176480:CCCAG:Cdonor_gain0.9900
2:26182961:AT:Aacceptor_gain0.9900

AlphaMissense

5557 scored. Top likely-pathogenic:

VariantProtein changeam_pathogenicity
2:26176383:T:CL51P1.000
2:26176403:T:AW58R1.000
2:26176403:T:CW58R1.000
2:26176419:C:AA63D1.000
2:26176428:T:CL66P1.000
2:26176446:T:AV72D1.000
2:26176449:T:AI73N1.000
2:26176451:G:AG74R1.000
2:26176451:G:CG74R1.000
2:26176451:G:TG74W1.000
2:26176452:G:AG74E1.000
2:26176452:G:TG74V1.000
2:26176467:T:AI79N1.000
2:26176467:T:GI79S1.000
2:26182972:T:CF87L1.000
2:26182973:T:CF87S1.000
2:26182973:T:GF87C1.000
2:26182974:C:AF87L1.000
2:26182974:C:GF87L1.000
2:26184282:T:CL145P1.000
2:26184300:T:AL151H1.000
2:26184300:T:CL151P1.000
2:26184491:T:CC215R1.000
2:26184495:T:AL216H1.000
2:26184495:T:CL216P1.000
2:26184500:T:CC218R1.000
2:26184502:C:GC218W1.000
2:26184534:T:AL229Q1.000
2:26184534:T:CL229P1.000
2:26184557:T:CF237L1.000

dbSNP variants (sampled 300 via entrez): RS1000037528 (2:26193496 C>T), RS1000056377 (2:26197949 C>T), RS1000185624 (2:26190517 C>T), RS1000272229 (2:26190360 T>A,C), RS1000297620 (2:26192008 G>A,T), RS1000426259 (2:26178890 G>GGAGGCGGAGGCGGAGGCA), RS1000475423 (2:26195956 C>T), RS1000567035 (2:26203173 G>A), RS1000578393 (2:26202820 A>G), RS1000758791 (2:26183799 C>A,G,T), RS1000969850 (2:26172622 C>A,G,T), RS1000980828 (2:26171623 G>A,C), RS1001105832 (2:26172010 C>A,G,T), RS1001167089 (2:26178011 G>A), RS1001192623 (2:26184169 G>A)

Disease associations

OMIM: gene MIM:617999 | disease phenotypes: MIM:609015, MIM:609016, MIM:614728

GenCC curated gene-disease

Mondo (5): metabolic disease (MONDO:0005066), mitochondrial trifunctional protein deficiency (MONDO:0012172), long chain 3-hydroxyacyl-CoA dehydrogenase deficiency (MONDO:0012173), mitochondrial trifunctional protein deficiency 1 (MONDO:0958181), Seckel syndrome 6 (MONDO:0013871)

Orphanet (2): Long chain 3-hydroxyacyl-CoA dehydrogenase deficiency (Orphanet:5), Mitochondrial trifunctional protein deficiency (Orphanet:746)

HPO phenotypes

0 total (0 of 0 shown, HPO-id order):

GWAS associations

0 associations (top):

MeSH disease descriptors (2)

DescriptorNameTree numbers
D008659Metabolic DiseasesC18.452
C566945Trifunctional Protein Deficiency With Myopathy And Neuropathy (supp.)

Drugs & pharmacology

Drug and pharmacology data

Is drug target: no

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

CTD chemical–gene interactions

14 total (human), top 14 by PubMed support.

ChemicalActions (top 5)PubMed papers
Benzo(a)pyreneaffects methylation, increases expression2
Estradiolincreases expression, affects cotreatment2
triphenyl phosphateaffects expression1
beta-lapachoneincreases expression1
2-methyl-2H-pyrazole-3-carboxylic acid (2-methyl-4-o-tolylazophenyl)amidedecreases expression1
(+)-JQ1 compounddecreases expression1
Air Pollutantsincreases abundance, increases expression1
Methotrexateincreases expression1
Thimerosaldecreases expression1
Urethanedecreases expression1
Aflatoxin B1increases expression1
Cadmium Chlorideincreases expression1
Acrylamidedecreases expression1
Particulate Matterincreases abundance, increases expression1

Clinical trials (associated diseases)

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

TrialPhaseStatusTitle
NCT00192621PHASE4COMPLETEDSeronegatives and Metabolic Abnormalities Protocol 2 (SAMA002): Study to Compare the Effect of Kaletra and Combivir® in HIV-Negative Healthy Subjects
NCT01443806PHASE4COMPLETEDCharacterisation of the Human Carboxylesterase 1 (CES1) Mutations in Thailand
NCT02645279PHASE4UNKNOWNComparison of Oral 30 % Dextrose and iv Midazolam Sedation During MRI in Neonates
NCT04653779PHASE4UNKNOWNA Clinical Trial to Evaluate the Preference Regarding Convenience of Medication and Efficacy/Safety of SUGAMET®XR Tablet 5/1000mg
NCT05084079PHASE4UNKNOWNDifferent Initial Insulin Dose Regimens on Time to Achieve Glycemic Targets and Treatment Safety in SIIT
NCT05855577PHASE4NOT_YET_RECRUITINGMotor Function Efficacy of Pharmacological Treatments Targeting Energy Metabolism, in Parkinson’s Patients
NCT06003153PHASE4RECRUITINGGLUCOSE-MGH: Genetic Links Understood Through Challenge With Oral Semaglutide Exposure at MGH
NCT06009653PHASE4WITHDRAWNEffects of Tirzepatide Plus Intensive Lifestyle Therapy on Body Weight and Metabolic Health in Latinos With Obesity
NCT00387114PHASE3TERMINATEDEffect of an Anti-Oxidant Treatment on Resistin Serum Levels.A Randomized Study
NCT00546052PHASE3COMPLETEDA 52 Week Study to Evaluate the Effects of Losartan With or Without HCTZ on Plasma Glucose, Metabolic Parameters, Blood Pressure in Hypertensive Patients With Metabolic Syndrome (0954A-331)
NCT00607373PHASE3COMPLETEDStudy to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
NCT00694109PHASE3COMPLETEDAn Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-Hypercholesterolemia
NCT00935766PHASE3TERMINATEDEffect of Fish Oil (Omega-3 Fatty Acids) on Arteries
NCT01286909PHASE3UNKNOWNEfficacy of Laflavon in Lowering Triglycerides and Raising High-Density Lipoprotein (HDL)
NCT01343680PHASE3TERMINATEDTrial of Two Central Venous Catheter (CVC) Flushing Schemes in Pediatric Hematology and Oncology Patients
NCT04371978PHASE3TERMINATEDEfficacy and Safety of Dipeptidyl Peptidase-4 Inhibitors in Diabetic Patients With Established COVID-19
NCT04809220PHASE3COMPLETEDA Study of Two Doses of Dulaglutide (LY2189265) in Japanese Patients With Type 2 Diabetes
NCT05260021PHASE3COMPLETEDA Study to Evaluate Tirzepatide (LY3298176) in Pediatric and Adolescent Participants With Type 2 Diabetes Mellitus Inadequately Controlled With Metformin or Basal Insulin or Both
NCT05425745PHASE3COMPLETEDEvaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.
NCT05505994PHASE3UNKNOWNThe Efficacy and Safety of DWP16001 in Combination With Metformin in T2DM Patients Inadequately Controlled on Metformin
NCT05691712PHASE3COMPLETEDA Study of Tirzepatide (LY3298176) in Chinese Participants With Type 2 Diabetes
NCT06568471PHASE3RECRUITINGA Study on Efficacy and Safety of HST101 in Chinese Patients with Hypercholesterolemia
NCT06672237PHASE3RECRUITINGA Phase 3 Study of NTLA-2001 in ATTRv-PN
NCT07613307PHASE3NOT_YET_RECRUITINGA Study of Orforglipron (LY3502970) in Participants With Type 2 Diabetes Who Observe Ramadan Fasting
NCT00001723PHASE2COMPLETEDSafety and Efficacy of Xenical in Children and Adolescents With Obesity-Related Diseases
NCT00119379PHASE2COMPLETEDEffectiveness of Nucleoside Supplementation or Switch to Tenofovir in Reversing Fat Loss in HIV Infected Adults
NCT00362180PHASE2COMPLETEDMeasure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
NCT00446264PHASE2COMPLETEDIslet Allotransplantation With Steroid Free Immunosuppression
NCT00477594PHASE2COMPLETEDOpen Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia
NCT00622765PHASE2COMPLETEDA Study of the Safety and Effectiveness of JNJ-16269110 (R256918) in Overweight and Obese Patients
NCT00707746PHASE2COMPLETEDSafety and Efficacy Study of ISIS 301012 (Mipomersen) Administration in High Risk Statin Intolerant Subjects
NCT01041677PHASE2COMPLETEDA Study of the Safety of R256918 in Obese Patients
NCT01135537PHASE2TERMINATEDPharmacokinetics of Thymoglobulin in Paediatric Haematopoietic Stem-cell Transplants
NCT01596699PHASE2TERMINATEDPilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation
NCT01927913PHASE2WITHDRAWNTreatment of Iron Overload Requiring Chelation Therapy
NCT01968720PHASE2COMPLETEDPilot Study To Assess CAT-2003 in Patients With Severe Hypertriglyceridemia
NCT02019667PHASE2COMPLETEDPhase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency
NCT02102737PHASE2COMPLETEDComparison of A New Technique of Measure of the Insulin Resistance By Scintigraphy With the Reference Technique
NCT02512679PHASE2TERMINATEDRelated Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells
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