资讯

MedPage Today on MSN1 天
Bone Health in Duchenne Muscular Dystrophy
Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to ...
REGENXBIO Publishes Preclinical Data Highlighting RGX-202’s Potential for Duchenne ...
REGENXBIO Inc. (NASDAQ:RGNX) is one of the biotech stocks to buy according to Wall Street analysts. On July 10, REGENXBIO ...
FDA’s Reversal on Gene Therapy Helps My Son
Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.
Muscular Dystrophy News2 天
Duchenne MD gene-editing therapy nets orphan drug designation
A mouse model study of Precision BioSciences’ gene-editing therapy PBGENE-DMD found it produced long-term functional ...
The American Journal of Managed Care2 天
FDA Recommends Sarepta Resume DMD Gene Therapy Shipments for Ambulatory Patients
Shipment of delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), will resume following a ...
Just The News3 天
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 ...
Lancashire Evening Post on MSN4 天
Cumbria family’s ‘Iron Will’ triathlon for son living with muscular dystrophy
On Thursday 28 August, Sam Taylor, 46, his wife Sue, 47, and daughter Beth, 17, from Crooklands in Cumbria will set off to ...
PharmiWeb4 天
MetrioPharm’s MP1032 Receives EMA Orphan Drug Designation in Duchenne Muscular Dystrophy
European Medicines Agency recognizes potential of lead compound MP1032 in addressing a high unmet medical need in Duchenne ...
FDA Probes Death Of 8-Year-Old Who Received Sarepta's Elevidys
FDA probes Elevidys after an 8-year-old's death in Brazil; Sarepta and Roche say the death was unrelated to gene therapy ...
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 opens probe into mysterious death linked to gene therapy
The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...
BioSpace2 天
FDA Issued Myeloma, Alzheimer’s Approvals in July but Rejected Two CGTs
The FDA greenlit multiple new drugs this month and issued some notable label expansions, including for Eli Lilly’s Kisunla.