About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is the most common muscular dystrophy, caused by mutations in the DMD gene encoding dystrophin. It affects primarily males, with progressive muscle wasting beginning in early childhood, loss of ambulation typically by age 12, and cardiac and respiratory involvement by the teens. Exon-skipping therapies (eteplirsen, golodirsen, viltolarsen, casimersen) are approved for specific mutation types. Gene therapy and CRISPR-based trials are actively recruiting. The DMD community is one of the most trial-active in rare disease.
Common Clinical Features
Clinical Trial Eligibility Tips
What to know before applying to Duchenne Muscular Dystrophy trials.
Mutation type (deletion, duplication, or point mutation) determines eligibility for exon-skipping drugs — get full DMD gene sequencing before reviewing trials.
The TREAT-NMD DMD Global Database is the largest DMD registry — registering increases your chances of being approached for new studies.
Steroid treatment (deflazacort or prednisone) is standard of care — confirm current steroid status when applying for trials as it affects washout requirements.
Patient Resources
Find recruiting Duchenne Muscular Dystrophy trials
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