News

An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...
The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...
REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...
The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...
Vinay Prasad has stepped down as head of the FDA’s biologics division just two months into the job, following growing ...
As gene therapy maker Sarepta Therapeutics tangles with FDA over its Duchenne treatment, patients and their families are ...
A new investigation into Duchenne muscular dystrophy (DMD) pathogenesis suggests that at least part of the muscle degeneration observed in DMD patients may result from the reduced production of ...
Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...
Sarepta will halt all shipments of Elevidys, its drug for Duchenne muscular dystrophy patients, by Tuesday evening.
The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...
A senior official at the US Food and Drug Administration has suddenly resigned after only three months in the job. Vinay Prasad’s departure followed controversy over a treatment for Duchenne muscular ...
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.