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SGSH

—N-Sulfoglucosamine Sulfohydrolase

Chromosome: 17

Location: 17:80206716-80220928

OMIM: 605270

Ensembl: ENSG00000181523

Official·OMIM:605270 Official·ENSG00000181523

Encodes heparan sulfate degrading enzyme. Mutations cause MPS IIIA (Sanfilippo A).

Treatment Roadmap — SGSH

Preclinical

Phase 1

Phase 2

Phase 3

Approved

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

Associated Diseases

Sanfilippo Syndrome Type A

Autosomal RecessivePrevalence: 1/100000lysosomal

Official·OMIM:252900 Official·ORPHA581

Summary

Clinical Trial

Approved Therapy

Registered Variant