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News-Medical.Net on MSN8m
Improving RNA drug delivery through intracellular traffic control
A recent study involving researchers from the University of Basel reveals that slowing down the intracellular transport of ...
WISH-TV on MSN8h
People with disabilities fear loss of coverage as new Medicaid rules take effect
Family fears losing Medicaid coverage, which they rely on for life-saving care. They face uncertainty due to new work ...
European Medical Journal1d
New Guidelines Aim to Transform Duchenne Orthopedic Care
New consensus guidelines outline best practices for orthopedic management in Duchenne muscular dystrophy, focusing on ...
Fierce Pharma5d
FDA investigates patient deaths after treatment with Sarepta's Duchenne gene therapy Elevidys
In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...
Sarepta Investor Sues After Duchenne Gene Therapy Patient Deaths
Sarepta Therapeutics Inc. misled investors about the safety and prospects for one of its Duchenne muscular dystrophy ...
WYDaily1d
Student with Duchenne Muscular Dystrophy Finding a Home at VPCC’s Historic Triangle Campus
One visit to Virginia Peninsula Community College’s Historic Triangle Campus last summer eased any concerns they might have had. Soon after entering the building, Nicholas and his mother, Liz, were ...
Edgewise falls after data for muscular dystrophy therapy
Edgewise Therapeutics (NASDAQ:EWTX) lost ~9% in the morning hours on Thursday after the company posted data from clinical ...
BioSpace3d
Edgewise Takes a Hit as FDA Blocks Accelerated Path for Becker Muscular Dystrophy Drug
The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for ...
FDA probes deaths of boys taking Sarepta Duchenne drug
The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...
Irish Examiner on MSN14h
Couple push for drug that slows down muscular dystrophy to be made available in Ireland
The drug Givinostat has been brought into parts of Britain. We want that too, but we need the government to pay for it,' said Una Ennis ...
STAT12dOpinion
Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys
Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...
No Accelerated FDA Approval For Edgewise's Rare Muscular Disease Drug; Shares Fall
Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...