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Case Report: The effect of early initiation of eteplirsen treatment on the cardiac and motor disease course in an individual with Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive muscle weakness. Here we provide a case report for a male aged 7 years and 3 months diagnosed with DMD after detecting a deletion of exons 48–50 of the DMD gene. The child initially presented with elevated transam...

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Principais autores: Yamen Masalha, Yoav Zehavi, Banan Musallam, Majd Khamaysi, Maali Abu Omer, Daniel L. Fink, Ronen Spiegel
Format: Artigo
Sprog:Inglês
Udgivet: Frontiers Media S.A. 2026-07-01
Serier:Frontiers in Pediatrics
Fag:
cardiomyopathy
case report
Duchenne muscular dystrophy
eteplirsen
exon skipping therapy
Online adgang:https://www.frontiersin.org/articles/10.3389/fped.2026.1672692/full
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