In an eagerly anticipated decision, the Food and Drug Administration Thursday approved the first gene therapy for muscular dystrophy. "Today's approval addresses an urgent unmet medical need and is an ...
Please enable JavaScript to read this content. Fifteen years ago, Scholar Muthamia was getting ready to welcome her second child. The pregnancy had no complications ...
Update: The US Food and Drug Administration has now approved the first gene therapy for Duchenne muscular dystrophy for children aged four and five. The one-time treatment, named Elevidys, will cost ...