MedPage Today on MSN2d
Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular DystrophyA young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
Sportschosun (English) on MSN13h
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 ...
Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...
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 ...
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...
Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...
REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...
The money is developing treatments for diabetes, cancer and Alzheimer's disease. It could also mean a cure for a Ridgefield ...
Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...
REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, a differentiated ...
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