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 ...
The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...
Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...
Sportschosun (English) on MSN8h
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 ...
RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...
A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular ...
Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature ...
Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...
A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...
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 ...
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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