Associated Papers
Comprehensive analysis of 2097 patients with dystrophinopathy based on a database from 2011 to 2021.
Zhao L et al.
Orphanet Journal of Rare Diseases, 2024
39182149 (PubMed )
Supplement: 13023_2024_3217_MOESM2_ESM.docx
A Comprehensive Analysis of 2013 Dystrophinopathies in China: A Report From National Rare Disease Center.
Tong YR et al.
Frontiers in Neurology, 2020
Genotypes and Phenotypes of DMD Small Mutations in Chinese Patients With Dystrophinopathies.
Wang L et al.
Frontiers in Genetics, 2019
30833962 (PubMed )
Supplement: Table_1.xlsx › Supplementary_table
Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.
Finkel RS et al.
Plos One, 2013
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