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 ...
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.
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 ...
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 ...
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, ...
CNN8y
Drug prices: Transparency demanded for $89,000 muscular dystrophy ... - CNN
Deflazacort, a newly FDA-approved drug for Duchenne muscular dystrophy, has an $89,000-a-year price tag. It has been put on hold because of an uproar over drug prices.
Medscape21h
EMA Says No to Duchenne Gene TherapyElevidys
The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.