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 comorbid sickle cell anemia. He has received eteplirsen treatment for 3 years with no clear adverse effects, including no increase in sickle cell crises.

Object Details

• Location: Deutsche Nationalbibliothek Frankfurt am Main
• Extent: Online-Ressource
• Language: Englisch

References and Relationships

• Bibliographic citation: Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia ; volume:14 ; number:3 ; year:2022 ; pages:396-399 ; extent:4 ; Case reports in neurology ; 14, Heft 3 (2022), 396-399 (gesamt 4)

Contributors, Places and Time

• Creator: Aiello, Gregory M. ; Cartwright, Michael Stephen

Further information

• DOI: 10.1159/000527358
• URN: urn:nbn:de:101:1-2023010423212726443448
• Rights: Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
• Last update: 15.08.2025, 7:21 AM CEST