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Pulmonary Function in Nonambulatory Patients with nmDMD from the STRIDE Registry and CINRG Duchenne Natural History Study: A Matched Cohort Analysis

A. Guenther Bernert,O. Nascimento,M. Tulinius,F. Buccella,I. Desguerre,J. Kirschner,E. Mercuri, F. Muntoni, J. Jiang,A. Kristensen,P. Trifillis,C. L. Santos,C. M. McDonald

Abstracts of the 46th Annual Meeting of the Society for NeuropediatricsNeuropediatrics（2021）

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Abstract
Background: STRIDE (Strategic Targeting of Registries and International Database of Excellence) is an ongoing, multicenter, observational registry providing data on ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) in routine clinical practice.
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nmdmd,stride registry,nonambulatory patients
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