Researchers combined advanced imaging techniques and theoretical physics to observe and explain how nanoclusters of the protein emerin form inside living cells. The study uncovers the molecular 'rules ...
Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...
RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...
Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
Sportschosun (English) on MSN1d
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 ...
Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely ...
Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease-- --Data Presented at the ...
Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature ...
A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...
A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...
Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.
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