Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by an X-linked recessive mutation to the dystrophin gene 1 that affects 1 in 5000 males worldwide 2. The absence of ...
New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...
New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...
A new treatment platform developed by researchers at the University of Texas MD Anderson Cancer Center was able to deliver messenger RNA (mRNA) of the full-length DMD gene into preclinical models of ...
Findings published in The American Journal of Pathology identify GLUD1 enzyme as a potential therapeutic target for muscle restoration through metabolic reprogramming, addressing clinically unmet need ...
Amsterdam, NL, February, 26. 2015 - The muscular dystrophies are known to target various muscle groups differentially. In addition to making limb muscles weak, muscular dystrophy (MD) can also lead to ...
Translational proof-of-concept studies for gene therapeutics often match the payload gene sequence to the preclinical animal model as this avoids immune responses that could compromise drug efficacy.
Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop ...
Researchers report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop a severe form of Duchenne ...
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