muscular dystrophy

The Food and Drug Administration approved the first gene therapy for Duchenne muscular dystrophy, but limited access to those ages four and five.

The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.

Although it’s still unclear what killed the 27-year-old, his death is raising questions about the experiment and the overall prospect of what one ethicist calls designer genetic therapies.

Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy.

H. Lee Sweeney, a world leader in research on neuromuscular diseases, has agreed to move to University of Florida next year to lead an institute…