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ASPA

—Aspartoacylase

Chromosome: 17

Location: 17:3472374-3503405

OMIM: 608034

Ensembl: ENSG00000108381

Official·OMIM:608034 Official·ENSG00000108381

Encodes aspartoacylase enzyme. Mutations cause Canavan disease.

Treatment Roadmap — ASPA

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

Canavan Disease

Autosomal RecessivePrevalence: 1/100000lysosomal

Official·OMIM:271900 Official·ORPHA141

Summary

Clinical Trial

Approved Therapy

Registered Variant