News

STAT1d
For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
Sarepta woes mount as Duchenne gene therapy knocked back in Europe
The European Medicines Agency determined testing failed to prove Elevidys’ benefit and issued a negative opinion that could ...
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 ...
STAT23h
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 ...
Sarepta pares gains as report suggests an ‘arduous’ path for Duchenne drug
Sarepta Therapeutics (NASDAQ:SRPT) faces an “arduous and treacherous path” to bring its gene therapy Elevidys back to market, ...
FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths
The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...
Sarepta Therapeutics crisis is huge blow to Duchenne families, company
Sarepta rebuffed a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular ...
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 ...
BioSpace21h
Sarepta Fallout Continues with EU’s Negative Opinion of Elevidys in Ambulatory Patients
The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne ...