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ARSA

—Arylsulfatase A

Chromosome: 22

Location: 22:50622754-50628177

OMIM: 607574

Ensembl: ENSG00000100299

Official·OMIM:607574 Official·ENSG00000100299

Encodes arylsulfatase A enzyme. Mutations cause metachromatic leukodystrophy.

Treatment Roadmap — ARSA

Preclinical

Phase 1

Phase 2

1 trial

Phase 3

1 trial

Approved

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

Associated Diseases

Metachromatic Leukodystrophy

Autosomal RecessivePrevalence: 1/100000lysosomal

Official·OMIM:250100 Official·ORPHA512

Summary

Clinical Trial

Approved Therapy

Registered Variant