This research article, published in the Journal of Comparative Effectiveness Research, provides results from a study investigating the efficacy of a mutation-specific therapy in delaying loss of ambulation (LoA) and respiratory function in patients with nonsense mutation DMD (nmDMD)
Publication: Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy (DMD) patients
MED-ALL-CORP-2200029 | October 2023
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