Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia

Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia

Eteplirsen is an antisense oligonucleotide used in the treatment of Duchenne muscular dystrophy (DMD). The safety of eteplirsen use in individuals with rare comorbid conditions is not known. We present the case of a 4-year-old boy with a DMD exon deletion amenable to treatment with eteplirsen and co...

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Bibliographic Details
Main Authors: Gregory M. Aiello, Michael Stephen Cartwright
Format: Article
Language:English
Published: Karger Publishers 2022-10-01
Series:Case Reports in Neurology
Subjects:
children
duchenne muscular dystrophy
treatment
mutation
Online Access:https://www.karger.com/Article/FullText/527358

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