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Medpage Today on MSN11h
Bone Health in Duchenne Muscular Dystrophy
An alternative to using standard glucocorticoids is the synthetic dissociative steroid vamorolone (Agamree), which was ...
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 ...
STAT8d
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 ...
Just The News3d
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 ...
MedPage Today on MSN4d
Shipments of Duchenne Gene Therapy to Resume After FDA Review of Patient Death
Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver ...
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 ...
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 ...
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 ...
Pharmabiz2d
Genethon gets UK MHRA nod to initiate pivotal phase 3 trial of GNT0004, a low-dose microdystrophin gene therapy for Duchenne muscular dystrophy
Genethon gets UK MHRA nod to initiate pivotal phase 3 trial of GNT0004, a low-dose microdystrophin gene therapy for Duchenne muscular dystrophy: Paris, France Wednesday, July 30, ...
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 ...
Nature18h
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 ...