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...

Descripción completa

Detalles Bibliográficos
Autores principales: Aiello, Gregory M., Cartwright, Michael Stephen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: S. Karger AG 2022
Materias:
Single Case − General Neurology
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
_version_ 1784868504185339904
author Aiello, Gregory M.
Cartwright, Michael Stephen
author_facet Aiello, Gregory M.
Cartwright, Michael Stephen
author_sort Aiello, Gregory M.
collection PubMed
description 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 comorbid sickle cell anemia. He has received eteplirsen treatment for 3 years with no clear adverse effects, including no increase in sickle cell crises.
format Online
Article
Text
id pubmed-9834637
institution National Center for Biotechnology Information
language English
publishDate 2022
publisher S. Karger AG
record_format MEDLINE/PubMed
spelling pubmed-98346372023-01-13 Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia Aiello, Gregory M. Cartwright, Michael Stephen Case Rep Neurol Single Case − General Neurology 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 comorbid sickle cell anemia. He has received eteplirsen treatment for 3 years with no clear adverse effects, including no increase in sickle cell crises. S. Karger AG 2022-10-24 /pmc/articles/PMC9834637/ /pubmed/36644004 http://dx.doi.org/10.1159/000527358 Text en Copyright © 2022 by The Author(s). Published by S. Karger AG, Basel https://creativecommons.org/licenses/by-nc/4.0/This article is licensed under the Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC). Usage and distribution for commercial purposes requires written permission.
spellingShingle Single Case − General Neurology
Aiello, Gregory M.
Cartwright, Michael Stephen
Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia
title Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia
title_full Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia
title_fullStr Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia
title_full_unstemmed Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia
title_short Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia
title_sort eteplirsen use in a boy with duchenne muscular dystrophy and sickle cell anemia
topic Single Case − General Neurology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9834637/
https://www.ncbi.nlm.nih.gov/pubmed/36644004
http://dx.doi.org/10.1159/000527358
work_keys_str_mv AT aiellogregorym eteplirsenuseinaboywithduchennemusculardystrophyandsicklecellanemia
AT cartwrightmichaelstephen eteplirsenuseinaboywithduchennemusculardystrophyandsicklecellanemia

Ejemplares similares