15 October 2016 - Approving an unproven drug sets a worrying precedent.
Duchenne muscular dystrophy is a horrible disease. Afflicting mainly boys, it weakens their muscles and eventually confines them to wheelchairs. In the end, typically when they are in their 20s, it kills them.
Patients and parents are understandably elated, therefore, at the decision taken last month by America’s drug agency, the FDA, to approve the first treatment for Duchenne. No one could fail to be moved by their campaign to win approval. At an FDA meeting this year one sufferer pleaded: “please don’t let me die early.”