A 1-year analysis from a natural history study in Chinese individuals with Duchenne muscular dystrophyResearch in context

• Main Authors: Xihua Li (Author), Junlan Lv (Author), Wenhua Zhu (Author), Siqi Hong (Author), Zhiqiang Wang (Author), Xingzhi Chang (Author), Ying Xu Gao (Author), Yangmei Zhou (Author), Caiping Jia (Author), Jia Fang (Author), Terrell A. Patterson (Author)
• Format: Book
• Published: Elsevier, 2024-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Summary: Background: Duchenne muscular dystrophy (DMD) is a disabling and life-threatening, X-linked recessive disorder caused by mutations in dystrophin. Natural history studies can inform the disease characteristics of DMD, and data from these studies can be used to plan and design clinical trials...