Boys with Duchenne muscular dystrophy (DMD) who cannot walk and have advanced scoliosis are at a higher risk of vertebral fractures due to significantly reduced bone mineral density (BMD), especially in the lumbar region of the spine, a study shows. Their low bone density may be associated with immobilization…
The FSHD Society has joined forces with the Critical Path Institute (C-Path) to facilitate the collection of data from people with facioscapulohumeral muscular dystrophy (FSHD) who were given a placebo in previous clinical trials. Provided by sponsors of FSHD trials, the data will be integrated into C-Path’s…
DT-DEC01, Dystrogen Therapeutics’ experimental chimeric cell therapy for Duchenne muscular dystrophy (DMD), appears generally safe and improved muscle strength and motor function in the first three boys enrolled in a Phase 1 clinical trial. Based on these positive results of the therapy’s low dose, with follow-up ranging from one…
An ion channel called Piezo1 regulates muscle stem cell (MuSC) states and the number of cells that respond quickly to muscle injury, promoting muscle repair, a study in mice shows. Notably, this ion channel was found to be poorly activated in a mouse model of Duchenne muscular dystrophy (DMD), and…
Santhera Pharmaceuticals has entered into an agreement with SEAL Therapeutics, a spin-off of the Biozentrum of the University of Basel, that is expected to further develop a potential gene therapy for LAMA2-deficient congenital muscular dystrophy (LAMA2 MD). The new company’s gene therapy technology builds on previous research…
Significantly higher levels of sphingolipids — a type of fatty molecule involved in inflammation, scarring, and cell death — are evident in the muscles of people with Duchenne muscular dystrophy and a DMD mouse model than in those of their healthy counterparts, a study shows. Notably, suppressing sphingolipid production in…
Cognitive problems seen in some Duchenne muscular dystrophy (DMD) patients may be associated with a shift toward the amyloidogenic pathway in memory-specific brain regions, according to a study in mouse models of the disease. The amyloidogenic pathway is a signaling cascade that leads to the formation of beta-amyloid, the…
The U.S. Food and Drug Administration (FDA) has approved Regenxbio’s request to launch a Phase 1/2 clinical trial in the U.S. evaluating the safety and efficacy of RGX-202, its experimental gene therapy for Duchenne muscular dystrophy (DMD). The study, called AFFINITY DUCHENNE and anticipated to start in…
The experimental oral therapy EDG-5506 is generally safe and results in rapid and profound reductions in the levels of muscle damage biomarkers in men with Becker muscular dystrophy (BMD), top-line data from a Phase 1b clinical trial show. “We believe the magnitude of reduction in multiple key biomarkers of muscle…
The U.K. Adult North Star Network (ANSN) has developed a set of guidelines for best care management of adults with Duchenne muscular dystrophy (DMD) based on consensus among experts in the field. The guidelines were outlined in a report, “Adult North Star Network (ANSN): Consensus Guideline…
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