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 ...

As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...

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

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to ...

The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.

European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...

EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory ...

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...

Duchenne muscular dystrophy (DMD) is an X-linked recessive myopathy that affects one in 3500 male births and which is caused by mutations in the dystrophin gene. The severe phenotype observed for ...

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 ...