GLA

Chr X

galactosidase alpha

Also known as: GALA

This gene encodes a homodimeric glycoprotein that hydrolyses the terminal alpha-galactosyl moieties from glycolipids and glycoproteins. This enzyme predominantly hydrolyzes ceramide trihexoside, and it can catalyze the hydrolysis of melibiose into galactose and glucose. A variety of mutations in this gene affect the synthesis, processing, and stability of this enzyme, which causes Fabry disease, a rare lysosomal storage disorder that results from a failure to catabolize alpha-D-galactosyl glycolipid moieties. [provided by RefSeq, Jul 2008]

GeneReviewsResearchGenerating clinical summary…

Clinical highlights

Gene-disease validity (ClinGen)
Fabry disease · XLDefinitivesufficient evidence for diagnostic panels
Interpreting a novel variant
Loss of function is the curated mechanism (Gene2Phenotype) and the gene is intolerant of it in the population — truncating, frameshift and canonical splice variants carry more prior weight here than missense.Curated gene-level mechanism — a prior for triage, not a per-variant call.
12
Active trials
589
Pubs (1 yr)
P/LP submissions
P/LP missense
0.18
LOEUF· LoF intol.
LOF
Mechanism· G2P
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GeneReview available — GLA
Authoritative clinical overview · Recommended first read
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Some data sources returned errors (1)

omim: Error: OMIM fetch failed: 429

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

LoF intolerant — likely haploinsufficient
LoF Constraint?
0.18LOEUF
pLI 0.997
Z-score 3.80
OE 0.00 (0.000.18)
Highly constrained

Highly LoF-intolerant (top ~10% of genes)

Missense Constraint?
1.88Z-score
OE missense 0.58 (0.490.69)
93 obs / 159.7 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios?
LoF OE?0.00 (0.000.18)
00.351.4
Missense OE?0.58 (0.490.69)
00.61.4
Synonymous OE?0.90
01.21.6
LoF obs/exp: 0 / 16.9Missense obs/exp: 93 / 159.7Syn Z: 0.56

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

GLA · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Fabry Disease

Fabry Disease Registry & Pregnancy Sub-registry

RECRUITING
NCT00196742Genzyme, a Sanofi CompanyStarted 2001-07-31
Fabry Disease

Natural History in Fabry Disease With IVS4+919G>A Mutations

ACTIVE NOT RECRUITING
NCT07506083Chinese University of Hong KongStarted 2022-10-20
Haematological MalignancySolid Tumour

DETERMINE (Determining Extended Therapeutic Indications for Existing Drugs in Rare Molecularly Defined Indications Using a National Evaluation Platform Trial) - Master Screening Protocol

RECRUITING
NCT05722886Phase PHASE2, PHASE3Cancer Research UKStarted 2023-03-01
AlectinibAtezolizumabEntrectinib
Fabry Disease

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

ACTIVE NOT RECRUITING
NCT06052800SanofiStarted 2023-09-13
Hemophilia A

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

ENROLLING BY INVITATION
NCT05768386BioMarin PharmaceuticalStarted 2023-01-01
Huntington Disease

A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

RECRUITING
NCT07326709Phase PHASE3Novartis PharmaceuticalsStarted 2026-03-24
Votoplam (blinded)Placebo
Transthyretin AmyloidosisATTR-CMATTRv-PN

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

RECRUITING
NCT06465810AstraZenecaStarted 2024-06-25
Treatment of transthyretin (ATTR) amyloidosis in observational study setting
Cerebral Small Vessel DiseasesCadasilHTRA1-Related Autosomal Dominant Cerebral Angiopathy

Taiwan Associated Genetic and Nongenetic Small Vessel Disease

RECRUITING
NCT05473637National Taiwan University HospitalStarted 2019-01-01
MRI
Short Chain Fatty Acids Concentration in StoolsGut Microbiota Diversity and CompositionBlood Lipid Profiles

Effects Of Dietary Fats on Gut Microbiota Composition and Metabolic Activity in Healthy Adults

RECRUITING
NCT07550023Phase NAUniversity of GlasgowStarted 2026-03-23
ButterCoconut oilExtra Virgin Olive Oil
Acute Respiratory Distress Syndrome (ARDS)

Clinical Evaluation of a Point of Care (POC) Assay to Identify Phenotypes in the Acute Respiratory Distress Syndrome

ACTIVE NOT RECRUITING
NCT04009330Queen's University, BelfastStarted 2019-11-22
POC Assay
Prostate Cancer

Study of Talazoparib With Enzalutamide in Men With DDR Gene Mutated mCSPC

ACTIVE NOT RECRUITING
NCT04821622Phase PHASE3PfizerStarted 2021-05-12
talazoparib plus enzalutamidePlacebo plus enzalutamide
Breast Cancer

Anti-Inflammatory Diet in BRC Patients on Aromatase Inhibitors

RECRUITING
NCT06214598Phase NAUniversity of BelgradeStarted 2024-03-01
SupplementAI dietPlacebo