Duchenne muscular dystrophy (DMD): genetic treatments

Evidence-based neurology checklist on duchenne muscular dystrophy (dmd): genetic treatments: 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…

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)

Drisapersen

Golodirsen

Viltolersen

Dilandistrogene moxeparvovec

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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