Disease

Dystrophinopathies / Duchenne muscular dystrophy1, 2

Gene

DMD

Inheritance XL
Current Treatments Eteplirsen, Casimersen and Golodirsen for exon skipping 51, 45 and 53, respectively. Vitolarsen has also been approved for exon 53 skipping. Translarna approved for nonsense variants.
Evidence for Treatment guideline3
Non-Molecular Testing Availability yes
Non-Molecular Test serum creatine kinase (CK) level4
Experimental / Investigational Therapies https://clinicaltrials.gov/ct2/results?cond=dmd&term=&cntry=&state=&city=&dist=
Last Updated: March 5, 2026
RX Genes
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