News

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 ...
European Medical Journal22h
New Guidelines Aim to Transform Duchenne Orthopedic Care
New consensus guidelines outline best practices for orthopedic management in Duchenne muscular dystrophy, focusing on ...
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
FDA has indicated that an Advisory Committee meeting is not required at this timeIn-person late-cycle review meeting ...
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 ...
Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
iBerkshires.com1d
Community Hero: Evan Butts
Although only living in the Berkshires for a few years, Evan Butts has already ingrained himself into the community. A server ...
BioSpace2d
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 investigates patient deaths after treatment with Sarepta's gene therapy
The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...
BioSpace4d
FDA Reviews Sarepta’s Elevidys After Second Patient Death
The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...
MedPage Today on MSN4d
FDA Probes Gene Therapy After Two Deaths
Duchenne muscular dystrophy is characterized by a mutation in the DMD gene that leads to a lack of dystrophin and muscle loss ...
Fierce Pharma4d
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, ...
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 ...