The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...

Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

A patient has died after receiving Sarepta Therapeutics’ approved gene therapy for Duchenne muscular dystrophy — the second fatality reported by the company in the past three months.

European regulators on Friday said that a Duchenne muscular dystrophy gene therapy should not be approved, further imperiling the future of the treatment that is facing increasing scrutiny in the ...

Regenxbio reported Monday the first clinical evidence showing improved muscle function in boys with Duchenne muscular dystrophy following treatment with its experimental gene therapy. The positive ...

LOS ANGELES (KABC) -- A first-of-its-kind gene therapy was just approved to treat Duchenne muscular dystrophy, a devastating condition affecting roughly 1 out of 3,500 to 5,000 boys. The game ...

A new paper, published in Gene Therapy, raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant ...

Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and a well-tolerated safety profile.

Second patient death reported with gene therapy for muscular dystrophy The U.S. Food and Drug Administration campus in Silver Spring, Md., is photographed, Oct. 14, 2015. Credit: AP/Andrew Harnik ...