Muscular dystrophy patients get first gene therapy

https://www.npr.org/sections/health-shots/2023/06/22/ [login to see] /muscular-dystrophy-patients-get-first-gene-therapy
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 important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options, that leads to a progressive deterioration of an individual's health over time," said Dr. Peter Marks, director of the FDA's Center for Biologics Evaluation and Research, in a statement.

But the agency rejected a request to make the treatment available to all children with Duchenne muscular dystrophy, the most common form of the incurable muscle disease, who could still walk. Instead, the agency restricted access to patients ages four and five until more evidence is available that the therapy is safe and effective.

The decision elicited mixed reactions. Parents of children suffering from the genetic disorder, advocates and some doctors and researchers welcomed the limited approval. But some were disappointed the treatment isn't being made more widely available right away.