Findings from the Longitudinal CINRG Becker Natural History Study.

Paula R Clemens,Heather Gordish-Dressman,Gabriela Niizawa,Ksenija Gorni,Michela Guglieri,Anne M Connolly,Matthew Wicklund,Tulio Bertorini,Jean Mah,Mathula Thangarajh,Edward C Smith,Nancy L Kuntz,Craig M McDonald,Erik Henricson, S Upadhyayula,Barry Byrne,Georgios Manousakis,Amy Harper,Susan Iannaccone,Utkarsh J Dang

Journal of neuromuscular diseases（2023）

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摘要

There is variable progression of outcomes resulting in significant heterogeneity of the clinical phenotype of Becker muscular dystrophy. Disease progression is largely manifest in adulthood. There are implications for clinical trial design revealed by this longitudinal analysis of a Becker natural history dataset.

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