- Rivera, Sarah;
- Jhamb, Sumit;
- Abdel-Hamid, Hoda;
- Acsadi, Gyula;
- Brandsema, John;
- Ciafaloni, Emma;
- Darras, Basil;
- Iannaccone, Susan;
- Konersman, Chamindra;
- Kuntz, Nancy;
- Parsons, Julie;
- Tesi Rocha, Carolina;
- Zaidman, Craig;
- Butterfield, Russell;
- Connolly, Anne;
- Mathews, Katherine;
- McDonald, Craig
INTRODUCTION: Duchenne muscular dystrophy (DMD) is a childhood onset muscular dystrophy leading to shortened life expectancy. There are gaps in published DMD care guidelines regarding recently approved DMD medications and alternative steroid dosing regimens. METHODS: A list of statements about use of currently available therapies for DMD in the United States was developed based on a systematic literature review and expert panel feedback. Panelists responses were collected using a modified Delphi approach. RESULTS: Among corticosteroid regimens, either deflazacort or prednisone weekend dosing was preferred when payer requirements do not dictate choice. Most patients with exon 51 skip-amenable mutations should be offered eteplirsen, before or with a corticosteroid. DISCUSSION: The options available for medical management of the motor symptoms of DMD are expanding rapidly. The choice of medical therapies should balance expected benefit with side effects.