Duchenne muscular dystrophy (DMD): genetic treatments

Written and reviewed by Dr Ibrahim Imam, Consultant Neurologist. Last reviewed .

Duchenne muscular dystrophy (DMD): genetic treatments. This is an approved exon skipping gene therapy. Fully referenced neurology checklist.

Eteplirsen (Exondys 51)

  • This is an approved exon skipping gene therapy
  • It restores dystrophin positive fibers
  • It improves walking distance
  • It reportedly slows down the rate of decline of ambulation

Ataluren (Translana)

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Drisapersen

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Golodirsen

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Viltolersen

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Dilandistrogene moxeparvovec

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References

  1. Bushby K, Muntoni F, Urtizberea A, Hughes R, Griggs R. Report of the 124th ENMC International Workshop. Treatment of Duchenne muscular dystrophy; defining the gold standards of management in the use of corticosteroids 2-4 April 2004, Naarden, The Netherlands. Neuromuscul Disord 2004; 14:526-534.
  2. Gloss D, Moxley RT 3rd, Ashwal S, Oskoui M. Practice guideline update summary: corticosteroid treatment of Duchenne muscular dystrophy: report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology 2016; 86:465-472.
  3. Syed YY. Eteplirsen: First global approval. Drugs 2016; 76:1699-1704.
  4. Mendell JR, Rodino-Klapac LR, Sahenk Z, et al; Eteplirsen Study Group. Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol 2013; 74:637-647.
  5. Randeree L, Eslick GD. Eteplirsen for paediatric patients with Duchenne muscular dystrophy: a pooled-analysis. J Clin Neurosci 2018; 49:1-6.
  6. And 21 more. Subscribe to see the full list

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