Since 2016, the FDA has approved a handful of therapies to treat Duchenne muscular dystrophy (DMD). But some uncertainty exists over their effectiveness, in addition to concerns about their costs. Additional products may be available soon, as the pipeline for the condition boasts several candidates.
According to the National Institute of Neurological Disorders and Stroke, muscular dystrophy consists of a group of more than 30 genetic diseases in which people lose muscle mass and strength over time. DMD, which mainly affects boys and is caused by a lack of dystrophin, is the most common form of the condition, accounting for about half the cases. Onset of the progressive disease is between 3 years old and 5 years old. Most boys with the condition will not be able to walk by the time they’re 12 years old. They will experience declining heart and lung functions and will require a respirator. People with the condition usually die in their 40s.