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The first gene therapy for muscular dystrophy has been approved for some kids

The U.S. Food and Drug Administration cleared a shortened version of a gene for a muscle protein to be used in 4- and 5-year-olds with muscular dystrophy. ....

United States , Douglas Ingram , Sharon Hesterlee , Drug Administration , Muscular Dystrophy Association , Sarepta Therapeutics , North America , Solid Biosciences ,

Sarepta Therapeutics announces FDA approval of first gene therapy to treat Duchenne muscular dystrophy

Sarepta Therapeutics Inc (NASDAQ:SRPT) said the US Food and Drug Administration (FDA) has accelerated approval for ELEVIDYS, its first-of-a-kind gene. ....

Jerry Mendell , Douglas Ingram , Sarepta Therapeutics Inc , Drug Administration , Gene Therapy , Nationwide Children ,

Sarepta's $3.2M Gene Therapy for DMD Just Got Approved. Why the Stock Is Down.

The $3.2 million one-time treatment is the first gene therapy approved to treat DMD, a progressive and fatal condition that manifests in early childhood. ....

Josh Nathan Kazis , Peter Marks , Douglas Ingram , Danielle Brill , Raymond James , Drug Administration , Sarepta Therapeutics , Sarepta Therapeutic , Wall Street , Biologics Evaluation , Bluebird Bio ,

US FDA approves Sarepta's gene therapy for rare muscular dystrophy in some kids | WKZO | Everything Kalamazoo

By Leroy Leo and Aditya Samal (Reuters) -The U.S. Food and Drug Administration on Thursday granted accelerated approval to Sarepta Therapeutics' first. ....

United States , Aditya Samal , Leroy Leo , Salveen Richter , Shinjini Ganguli , Raghav Mahobe , Douglas Ingram , Sarepta Ingram , Drug Administration On , National Organization For Rare Disorders , Goldman Sachs , Drug Administration , Sarepta Therapeutic , National Organization ,

US FDA approves Sarepta's gene therapy for rare muscular dystrophy in some kids

The U.S. health regulator has granted accelerated approval to Sarepta Therapeutics' first-of-its-kind gene therapy for Duchenne muscular dystrophy (DMD), an inherited progressive muscle-wasting disorder that almost always affects young boys. Sarepta said on Thursday the Food and Drug Administration had approved the treatment for children aged between 4 and 5 years who can walk. It was initially seeking approval for all DMD patients who can walk. ....

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