The experience of using ataluren in Duchenne muscular dystrophy in Moscow: first results

The experience of using ataluren in Duchenne muscular dystrophy in Moscow: first results

Duchenne muscular dystrophy (DMD) is an X-linked inherited neuromuscular disorder typically manifesting in boys aged 2–5 years, characterized by a progressive course. According to natural disease progression data, individuals with DMD typically lose the ability to walk independently by the age of 13...

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Bibliographic Details
Main Authors: T. N. Kekeeva, N. L. Pechatnikova, I. P. Vitkovskaya, V. S. Kakaulina, N. A. Krasnoschekova, Yu. E. Martynenko
Format: Article
Language:Russian
Published: ABV-press 2024-01-01
Series:Нервно-мышечные болезни
Subjects:
duchenne muscular dystrophy
pathogenic therapy
ataluren
translarna
Online Access:https://nmb.abvpress.ru/jour/article/view/573
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