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Plain Language Summary: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.pdf

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posted on 2021-11-18, 13:43 authored by Craig McDonaldCraig McDonald, Francesco MuntoniFrancesco Muntoni, Vinay Penematsa, Joel Jiang, Allan Kristensen, Francesco Bibbiani, Elizabeth GoodwinElizabeth Goodwin, Heather Gordish-Dressman, Lauren Morgenroth, Christian WernerChristian Werner, James Li, Richard Able, Panayiota Trifillis, Már Tulinius, On behalf of the Study 019 & CINRG Duchenne Natural History Investigators

Plain Language Summary: Long-term effect of ataluren in patients with a specific type of genetic mutation leading to Duchenne muscular dystrophy

Funding

This work was supported by PTC Therapeutics. Study 019 was sponsored by PTC Therapeutics. The CINRG DNHS was funded by grants from the US Department of Education/NIDRR (#H133B031118, #H133B090001), the US Department of Defense (#W81XWH-09-1-0592), the National Institutes of Health (#UL1RR031988, #U54HD053177, #UL1RR024992, #U54RR026139, #2U54HD053177, #G12RR003051, #1R01AR061875, #RO1AR062380) and Parent Project Muscular Dystrophy.

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

Journal of Comparative Effectiveness Research

Categories

Neurology and neuromuscular diseases

Keywords

ataluren efficacy dystrophin loss of ambulation nonsense mutation Duchenne muscular dystrophy respiratory function Study 019 Neurology and Neuromuscular Diseases

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CC BY-NC-ND 4.0

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