Transferring a healthy version of the Large1 gene into adult mice with severe congenital muscular dystrophy restored the function of muscles involved in movement and breathing, a study found. The use of this gene therapy translated into an extended lifespan for the mice, which had congenital muscular dystrophy —…

Edgewise Therapeutics announced the opening of a Phase 1 clinical trial to assess the safety and tolerability of EDG-5506, its investigational oral treatment for Becker muscular dystrophy (BMD). The study (NCT04585464) is now enrolling up to 152 healthy volunteers and adults with BMD, ages 19 to 55, at…

The absence of a signaling protein leads to immature muscle cells being unable to fuse together to promote muscle tissue growth, a study shows. University of Louisville researchers said lack of the MyD88 signaling protein prevented immature muscle cells known as myoblasts from fusing. When the protein’s levels are normal,…

Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene expressed in differentiated myofibers. Researchers at The Ottawa Hospital and the University of Ottawa recently discovered that muscle stem cells also express dystrophin, affecting the generation of functional muscle fibers. Their discovery could revolutionize understanding of the disease and eventually…

Cellular models can elucidate molecular mechanisms underlying several diseases, reducing animal usage in biomedical sciences and following the guiding principles for animal testing. Now, researchers at the Karolinska Institute and at the Karolinska University published a study entitled “Primary Murine Myotubes as a Model for Investigating Muscular…

A study recently published in the journal Nature Biotechnology revealed a new technique to generate muscle fibers in a laboratory setting, offering a better model to study muscular diseases including Duchenne muscular dystrophy (DMD). The study is entitled “Differentiation of pluripotent stem cells to…