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Research Article
18 November 2021

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

Authors: Craig M McDonald https://orcid.org/0000-0002-8779-3220 [email protected], Francesco Muntoni https://orcid.org/0000-0002-9102-5232, Vinay Penematsa, Joel Jiang, Allan Kristensen, Francesco Bibbiani, Elizabeth Goodwin, … Show All … , Heather Gordish-Dressman https://orcid.org/0000-0002-2330-5427, Lauren Morgenroth, Christian Werner, James Li, Richard Able, Panayiota Trifillis, and Már TuliniusAuthor Info & Affiliations
Volume 11, Number 3
https://doi.org/10.2217/cer-2021-0196
Plain Language Summary
PDFFormats

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. ClinicalTrials.gov registration: NCT01557400.

Supplementary Material

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