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DMD

—Dystrophin

Chromosome: X

Location: X:31097677-33339609

OMIM: 300377

Ensembl: ENSG00000198947

Official·OMIM:300377 Official·ENSG00000198947

Encodes the muscle structural protein dystrophin. Mutations cause Duchenne/Becker muscular dystrophy.

Treatment Roadmap — DMD

Preclinical

Phase 1

9 trial

Phase 2

8 trial

Phase 3

10 trial

Approved

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

Associated Diseases

Duchenne Muscular Dystrophy

X-Linked RecessivePrevalence: ~300Kneuromuscular

Official·OMIM:310200 Official·ORPHA98896

Becker Muscular Dystrophy

X-Linked RecessivePrevalence: 1/18000neuromuscular

Official·

DMD

Treatment Roadmap — DMD

Associated Diseases

Summary