In the last few years, the Duchenne muscular dystrophy (DMD) therapeutic landscape has undergone a dramatic evolution. More therapies are in clinical development today than ever before, each one aiming to treat DMD in a different way in order to halt or slow the disease’s devastating effects. This September marks the two-year anniversary of the . . .
BioMarin Pharmaceutical announced May 31 that it is discontinuing development of its exon skipping drug drisapersen. The company recently withdrew its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting that indicated the CHMP intended to issue a negative . . .
When a family first receives a muscular dystrophy diagnosis, the news can be overwhelming. Families suddenly find themselves faced with the need to learn about the disease, how to manage their loved one’s care, how to find the right resources, and how to plan for what lies ahead. Even those who have traveled this road . . .
