Sportschosun (English) on MSN38m
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 ...
MedPage Today on MSN1d
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 ...
Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...
Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...
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 ...
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...
A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...
Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...
REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...
New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ...
Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease-- --Data Presented at the ...
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