Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients

Craig M. McDonald, Francesco Muntoni, Vinay Penematsa, Joel Jiang, Allan Kristensen, Francesco Bibbiani, Elizabeth Goodwin, Heather Gordish-Dressman, Lauren Morgenroth, Christian Werner, James Li, Richard Able, Panayiota Trifillis, Már Tulinius

Research output: Contribution to journalArticlepeer-review

1 Scopus citations

Abstract

Aim: We investigated the effect of ataluren plus standard of care (SoC) on age at loss of ambulation (LoA) and respiratory decline in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) versus patients with DMD on SoC alone. Patients & methods: Study 019 was a long-term Phase III study of ataluren safety in nmDMD patients with a history of ataluren exposure. Propensity score matching identified Study 019 and CINRG DNHS patients similar in disease progression predictors. Results & conclusion: Ataluren plus SoC was associated with a 2.2-year delay in age at LoA (p = 0.0006), and a 3.0-year delay in decline of predicted forced vital capacity to <60% in nonambulatory patients (p = 0.0004), versus SoC. Ataluren plus SoC delays disease progression and benefits ambulatory and nonambulatory patients with nmDMD.

Original languageEnglish (US)
Pages (from-to)139-155
Number of pages17
JournalJournal of Comparative Effectiveness Research
Volume11
Issue number3
DOIs
StatePublished - Feb 2022

Keywords

  • ataluren
  • dystrophin
  • efficacy
  • loss of ambulation
  • nonsense mutation Duchenne muscular dystrophy
  • respiratory function
  • Study 019

ASJC Scopus subject areas

  • Health Policy

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