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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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Detalles Bibliográficos
Autores principales: Aiello, Gregory M., Cartwright, Michael Stephen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: S. Karger AG 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9834637/
https://www.ncbi.nlm.nih.gov/pubmed/36644004
http://dx.doi.org/10.1159/000527358