MDA 2020 abstract: Pulmonary function in non-ambulatory patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) from the STRIDE Registry and CINRG Duchenne Natural History Study: A matched cohort analysis
Get a brief comparison of pulmonary function decline between two matched cohorts of patients with Duchenne muscular dystrophy (DMD) in our abstract presented at MDA 2020
The objectives and approach taken in a matched cohort analysis comparing pulmonary function decline in non-ambulatory nmDMD patients treated with a mutation-specific DMD therapy plus standard of care (SoC; corticosteroid or palliative therapies) and matched patients with DMD receiving SoC alone (Cooperative International Neuromuscular Research Group [CINRG]) Duchenne Natural History Study are presented in this abstract
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