Positive experience in treating patients with Duchenne muscular dystrophy caused by a nonsense mutation: family clinical case

Duchenne muscular dystrophy is a severe genetic disease caused by mutations in the dystrophin gene (DMD), which lead to a significant decrease or complete absence of the protein of the same name in muscle fibers. The disease manifests itself in boys from the age of 2–5 years in the form of progressi...

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Bibliographic Details
Published in:Нервно-мышечные болезни
Main Authors: L. S. Kraeva, E. V. Fadeeva
Format: Article
Language:Russian
Published: ABV-press 2024-09-01
Subjects:
Online Access:https://nmb.abvpress.ru/jour/article/view/624