The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

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 ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...

When Chase Finazzo was just a few years old, his parents noticed Chase was pretty clumsy. But they didn't think it was anything serious. "He would fall a lot. Not like a lot a lot. But he had trouble ...

The Muscular Dystrophy Association (MDA) celebrates today's announcement that the U.S. Food and Drug Administration (FDA) has approved a High Dose Regimen of SPINRAZA(R) (nusinersen) for the treatment ...

The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval on Thursday despite concerns from some government scientists about the treatment's ability to help ...

The Food and Drug Administration Thursday approved the first gene therapy treatment for Duchenne Muscular Dystrophy, a progressive disorder that typically robs boys of their ability to walk around age ...

Susan and Chris Finazzo have enrolled their sons Dylan and Chase in a study of gene therapy for Duchenne muscular dystrophy. The experimental treatment is still being studied but researchers hope it ...

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. The creation of the ...