News

Two boys died after a gene therapy. This family won’t give up hope.
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients died, the drug’s manufacturer halted dosing under pressure from the FDA.
MIT Technology Review1d
The deadly saga of the controversial gene therapy Elevidys
The drug, which was designed to treat Duchenne muscular dystrophy, has been linked to the deaths of two teenage boys.
STAT1d
Europe moves to reject embattled Duchenne muscular dystrophy gene therapy
European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...
MedPage Today4d
Drugmaker Refuses FDA's Request to Pull Gene Therapy Tied to Patient Deaths
The agency also revoked Sarepta's platform technology designation for AAVrh74 Friday and issued a safety communication saying ...
Health Beat: Duchenne muscular dystrophy
Genetic testing could not confirm Duchenne muscular dystrophy, but a muscle biopsy at UCLA did. "He could see that he was ...
USA Today2y
Duchenne muscular dystrophy treatments, gene therapy spark hope - USA TODAY
Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...
MedCity News2y
FDA Approves First Gene Therapy for Duchenne Muscular Dystrophy
The Duchenne muscular dystrophy drugs now available only treat the symptoms of the rare muscle-wasting disorder. On Thursday, the FDA approved a Sarepta Therapeutics gene therapy, making it ...
TipRanks on MSN11h
FDA investigating death of child who received Sarepta’s Elevidys
The U.S. FDA is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta (SRPT) Therapeutics gene therapy for Duchenne ...