News

CBS Texas on MSN2h
North Texas teen battles rare disease with creativity and courage
A North Texas family is sharing a son's journey with Duchenne muscular dystrophy, a rare and progressive muscle-wasting ...
STAT7h
Disagreements over Duchenne therapy, management style may have led to ouster of key FDA official
The ouster of the FDA's chief regulator of cell and gene therapies came immediately after a disagreement with her boss over a ...
Capricor Therapeutics Announces Positive 4-Year Data from HOPE-2 Open-Label Extension Study of Deramiocel in Duchenne Muscular Dystrophy
Four-year data show preservation of cardiac function, including LVEFSkeletal muscle disease progression continues to slow ...
Second patient death reported with gene therapy for muscular dystrophy
Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...
The American Journal of Managed Care4d
Data on Duchenne Muscular Dystrophy Treatment From MDA 2025
Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows ...
MedPage Today on MSN4d
Second Patient Dies After Gene Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy patient died after being treated with the gene therapy delandistrogene moxeparvovec (Elevidys), ...
Biotech pauses trial after second patient death linked to gene therapy
Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular ...
Capricor Therapeutics Announces Orphan Drug Designation for Becker Muscular Dystrophy and Regulatory Progress for Duchenne Muscular Dystrophy Program
U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s ...
BioPharma Dive4d
Second Duchenne patient dies after receiving Sarepta gene therapy
Another person treated with Elevidys suffered acute liver failure, leading Sarepta to discontinue treatment in certain ...
Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio cream
Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio ...
Sarepta reports second patient death after treatment with Duchenne gene therapy
Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for ...
Major League Baseball1d
HOPE Week helps Yankee fans with DMD live out broadcasting dreams
NEW YORK – Carmine and Vincent Gagliano, two brothers from Brooklyn, were living the dream on Thursday at Yankee Stadium. It was like watching two kids in the candy store. They love the Yankees that ...