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

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.Case Rep Neurol 2022;14:404 –407
Source: Case Reports in Neurology - Category: Neurology Source Type: research