Objective To make a correctdiagnosis in theearly stage and avoid misdiagnosis, we analysedclinical data of patients with Duchennetype muscular dystrophy(DMD).
Even with improved mechanical breathing assistance, people who have Duchenne muscular dystrophy the most common type of muscular dystrophy usually die of respiratory failure before they reach age 40.
One big question: would the limited data Sarepta has so far collected from a very small clinical trial of its drug eteplirsen, a treatment for Duchenne muscular dystrophy that is caused by a very specific type of mutation, be enough to warrant filing with the Food and Drug Administration and perhaps even gain approval?