Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...
Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature ...
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 ...
14don MSN
A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular ...
The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...
GlobalData on MSN2d
Avidity’s del-zota elicits 25% increase in dystrophin in DMD patientsThe company plans to submit a biologics licence application to the US Food and Drug Administration later this year.
Sportschosun (English) on MSN9h
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 ...
A new discovery about how tiny protein clusters form in cells could pave the way for treatments for Emery-Dreifuss muscular ...
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 ...
Duchenne muscular dystrophy (DMD ... Blood tests might reveal elevated creatine kinase levels, a sign of muscle cell damage. A muscle biopsy (tissue sample) is usually taken from the thigh ...
Researchers have developed a strategy to treat muscular dystrophy, which uses nanoparticles as vehicles to transport therapeutical microRNAs to muscle stem cells. Once inside the muscle stem cells ...
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