A U.S. Food and Drug Administration (FDA) advisory board concluded that evidence supporting the effectiveness of Translarna (ataluren), by PTC…
Articles by Magdalena Kegel
PTC Therapeutics shared additional data from a Phase 3 trial of Translarna (ataluren), showing that the treatment slightly slowed…
The first patients with Duchenne muscular dystrophy (DMD) have been enrolled into the U.K.’s Early Access to Medicines Scheme (EAMS)…
Parent Project Muscular Dystrophy (PPMD) will host a one-hour webinar at 2 p.m. Eastern Time on Wednesday, Sept. 6, that…
Treadmill training using a voluntary-driven exoskeleton might be a safe approach to improve movement and exercise capacity in patients with…
The chemical structure of a drug can make the entire difference between a success and a failure in drug development.
The U.S. Food and Drug Administration has approved Capricor Therapeutics’ plan for further development of its CAP-1002 stem cell…
Long after it arrived elsewhere, Emflaza (deflazacort) became the first corticosteroid that the U.S. Food and Drug Administration approved…
Healthcare costs for patients with Duchenne muscular dystrophy (DMD) are about 10 times those of healthy people, and increase as…
Using glucocorticoid steroids once a week instead of once a day to slow the progression of muscular dystrophy (MD) could prevent…