Exon Skipping Therapy.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 27863231.
- Also identified by DOI 10.1016/j.cell.2016.10.050.
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Abstract
Exondys 51 is the first therapy for Duchenne muscular dystrophy (DMD) to have been granted accelerated approval by the FDA. Approval was granted based on using dystrophin expression as a surrogate marker. Exondys 51 targets DMD exon 51 for skipping to restore the reading frame for 13% of Duchenne patients.
Medical subject headings
- Dystrophin
- Genetic Therapy
- Muscular Dystrophy, Duchenne