Author Archives: Marta Figueiredo PhD

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…

Upper limb strength, function, and muscle fat fraction continuously worsen over time in Duchenne muscular dystrophy (DMD) patients with mutations amenable to exon 53 skipping, according to three-year data from a natural history study. Notably, this progression of upper limb involvement was assessed reliably with strength, function, and muscle…

A Phase 1 clinical trial evaluating Edgewise Therapeutics’ experimental oral therapy EDG-5506 in healthy volunteers and men with Becker muscular dystrophy (BMD) has dosed the first BMD patients. The study (NCT04585464) is currently recruiting up to eight men, ages 18 to 55, with BMD at its…