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...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
Karger Publishers
2022-10-01
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| Series: | Case Reports in Neurology |
| Subjects: | |
| Online Access: | https://www.karger.com/Article/FullText/527358 |