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Supplementary materials: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.docx

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posted on 2021-11-18, 13:33 authored by Craig 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

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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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Keywords

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

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