Dystrophin restoration or replacement aims to treat the underlying cause of Duchenne which is the lack of dystrophin, the protein that provides stability to the muscles. This panel will feature companies developing exon skipping and nonsense mutation read-through therapies that aim to restore dystrophin production.
Panelists:
PTC Therapeutics (Richard A. Able Jr., PhD, DMD Therapeutic Area Lead, Global Medical Affairs)
Daiichi-Sankyo (Niwata Yuichiro, M.S., R.Ph. Global Team Leader DS 5141 Exon Skipping Program)
Sarepta (Olga Mitelman, MD, Head of US Medical Affairs)
NS Pharma (Leslie Magnus, MD, VP Medical Affairs)
by: Parent Project Muscular Dystrophy
