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 dys- trophy (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 treat- ment 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.