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GBA1

—Glucosylceramidase Beta

Chromosome: 1

Location: 1:155231270-155245178

OMIM: 606463

Ensembl: ENSG00000177628

Official·OMIM:606463 Official·ENSG00000177628

Encodes lysosomal glucocerebrosidase enzyme. Mutations cause Gaucher disease.

Treatment Roadmap — GBA1

Preclinical

Phase 1

3 trial

Phase 2

3 trial

Phase 3

5 trial

Approved

Clinical trials have approximately 10% success rate. This roadmap shows the current status, not guaranteed outcomes.

Associated Diseases

Gaucher Disease

Autosomal RecessivePrevalence: ~10Kmetabolic

Official·OMIM:230800 Official·ORPHA355

Summary

Clinical Trial

Approved Therapy

Registered Variant