natural history

Exondys 51 Superior to Glucocorticoids in Preserving Respiratory Health of Duchenne Boys, Analysis Shows

Treatment with Exondys 51 (eteplirsen) significantly slows respiratory decline in boys with Duchenne muscular dystrophy (DMD), including those with more advanced disease, compared to standard glucocorticoid therapy, an analysis of clinical trials shows. The study “Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy”…

Utah Researcher Receives Grant to Study Stages of Congenital Myotonic Dystrophy

A University of Utah medical professor has received a $600,000 Muscular Dystrophy Association grant to conduct a clinical trial aimed at understanding how congenital myotonic dystrophy develops over time. The three-year grant will allow Dr. Nicholas Johnson, an assistant professor of neurology, pediatrics, and pathology, to study what scientists call the natural history…