After Sarepta reported the death of a patient who had recently taken the gene therapy Elevydis, patient advocacy group Parent ...
U.S. Sen. Richard Blumenthal and researchers raise the alarm on how a federal medical research funding cap could impact "life ...
The money is developing treatments for diabetes, cancer and Alzheimer's disease. It could also mean a cure for a Ridgefield ...
HealthDay on MSN14h
Sarepta’s Gene Therapy Under Scrutiny After Patient DeathA patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death ...
US gene therapy company Regenxbio has reported new, positive interim data from two additional patients in the Phase I/II ...
Regenxbio has reported positive interim data from the Phase I/II portion of its Affinity Duchenne study, which is examining ...
The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...
NEW YORK, NY / ACCESS Newswire / March 20, 2025 / Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on ...
ROCKVILLE, MD, USA I March 19, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase ...
Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced it ...
Announced the U.S. FDA has accepted our Biologics License Application (BLA) seeking full approval of deramiocel for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathyBLA granted priorit ...
Sportschosun (English) on MSN11h
New Treatment Strategy for Rare Disease Dutsenic Muscle Dystrophy...EZH2 inhibitor steroid effective in combinationKorean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback