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Bone Health in Duchenne Muscular Dystrophy
An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...
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The story of Sarepta's Duchenne gene therapy
The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The ...
REGENXBIO Publishes Preclinical Data Highlighting RGX-202’s Potential for Duchenne Muscular Dystrophy
REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...
Just The News2d
FDA allows distribution of muscular dystrophy drug again after public criticism
The agency’s now-reversed decision to halt distribution of a gene therapy for children suffering from muscular dystrophy ...
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FDA Official Who Halted Sarepta’s Gene Therapy Resigns Amid Backlash Over Access To Duchenne Treatment
Vinay Prasad has stepped down as head of the FDA’s biologics division just two months into the job, following growing ...
Parents call on FDA for path forward after Duchenne's gene therapy pause | Morning in America
The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...
STAT7d
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 ...
STAT3d
FDA permits use of Sarepta Therapeutics’ Duchenne therapy in younger patients after short-lived halt
The FDA said it was clearing the way for Sarepta Therapeutics to resume shipments of its gene therapy for Duchenne muscular ...
Fierce Pharma3d
FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients
Monday, the FDA said Sarepta may resume treating ambulatory DMD patients with Elevidys. The recommendation comes just 10 days ...
The BMJ1d
Vinay Prasad leaves FDA after less than three months amid Duchenne gene therapy controversy
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 ...
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 ...