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MedPage Today7h
Bone Health in Duchenne Muscular Dystrophy
An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...
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 ...
Amazon S3 on MSN2d
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 ...
Stocktwits on MSN2d
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 ...
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 ...
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 ...
Fierce Pharma4d
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 ...
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 ...
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 ...
STAT4d
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 ...
Nature13h
NO vascular control in Duchenne muscular dystrophy - Nature
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 ...