Article
Longitudinal motor function in proximal versus distal DMD pathogenic variants.
Muscle & nerve - 1 Oct 2021
Thangarajh Mathula, Bello Luca, Gordish-Dressman Heather
Abstract excerpt
INTRODUCTION/AIMS: There is considerable heterogenicity in clinical outcomes in Duchenne muscular dystrophy (DMD). The aim of this study was to assess whether dystrophin gene (DMD) pathogenic variant location influences upper or lower extremity motor function outcomes in a large prospective cohort. METHODS: We used longitudinal timed and quantitative motor function measurements obtained from 154 boys with DMD...
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