Duchenne muscular dystrophy (DMD) is a common progressive muscular disorder. It is an X-chromosome-linked disorder that gradually weakens various muscle groups including those supporting the heart, ...
- New preclinical data for the PBGENE-DMD final clinical candidate demonstrates an increase in dystrophin positive muscle cells across key muscle types, potentially driven by editing of muscle ...
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 ...
- Potential first-in-class gene editing approach designed for dystrophin gene correction leading the body to produce a functional dystrophin protein applicable for majority of DMD patients (up to ~60% ...
Duchenne muscular dystrophy, or DMD, is an X-linked progressive and severe neuromuscular disease caused by the lack of functional dystrophin, which causes atrophy and weakness in skeletal and heart ...
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 ...
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder affecting roughly 1 in 3,500 males. DMD is due to mutations in the dystrophin gene, which encodes for an exceptionally large 427 kD ...
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