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Updated: Mar 23, 2026

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Clinical utility of serum biomarkers in Duchenne muscular dystrophy
Yetrib Hathout1, Haeri Seol1, Meng Hsuan J Han1
1Center for Genetic Medicine, Children's National Healthy System, Washington, DC USA.
Abstract:
Assessments of disease progression and response to therapies in Duchenne muscular dystrophy (DMD) patients remain challenging. Current DMD patient assessments include complex physical tests and invasive procedures such as muscle biopsies, which are not suitable for young children. Defining alternative, less invasive and objective outcome measures to assess disease progression and response to therapy will aid drug development and clinical trials in DMD. In this review we highlight advances in development of non-invasive blood circulating biomarkers as a means to assess disease progression and response to therapies in DMD.
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