Article
Genotype characterization and delayed loss of ambulation by glucocorticoids in a large cohort of patients with Duchenne muscular dystrophy.
Orphanet journal of rare diseases - 28 Apr 2021
Zhang Shu, Qin Dongdong, Wu Liwen, Li Man, Song Lifang, Wei Cuijie, Lu Chunling, Zhang Xiaoli, Hong Siqi, Ma Mingming, Wu Shiwen
Abstract excerpt
BACKGROUND: Duchenne muscular dystrophy (DMD) is the most common genetic muscle disease in human. We aimed to describe the genotype distribution in a large cohort of Chinese DMD patients and their delayed loss of ambulation by glucocorticoid (GC) treatments. This is to facilitate protocol designs and outcome measures for the emerging DMD clinical trials. RESULTS: A total of 1163 patients with DMD were recruited...
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