Marisa Wexler, MS,  senior science writer—

A new clinical trial is seeking about 80 people with limb-girdle muscular dystrophy type 2i (LGMD2i) to gather information on this disease’s progression that may help in advancing potential therapies and in establishing a biomarker. The observational study (NCT04202627) is now enrolling patients, ages 10 to 65, at…

Supplementing the diet with the amino acid L-arginine may improve gut function in people with Duchene Muscular Dystrophy (DMD), a study in mice suggests. The study, “Spatiotemporal Mapping Reveals Regional Gastrointestinal Dysfunction in Mdx Dystrophic Mice Ameliorated by Oral L-arginine Supplementation,” was published in the …

Addressing changes in cellular metabolism and oxidative damage may lead to muscle regeneration in Duchenne muscular dystrophy and Becker muscular dystrophy, a study has found. The study, “Comparative proteomic analyses of Duchenne muscular dystrophy and Becker muscular dystrophy muscles: changes contributing to preserve muscle…

Translarna (ataluren) significantly improves physical fitness in people with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), a study examining multiple endpoints across two trials shows. The study used a statistical strategy for analyzing multiple trial endpoints together, a tactic that could improve the interpretation…

A new project called BIND — Brain Involvement  iN  Dystrophinopathies — seeks to better characterize how the brain is affected in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), and to find potential ways to treat neurological complications in people with those disorders. The European Union awarded…

Blocking the protein receptor IGF2R could promote muscle regeneration and improve muscle strength in people with Duchenne muscular dystrophy (DMD), a study in mice suggests. The study, “Blockade of IGF2R improves muscle regeneration and ameliorates Duchenne muscular dystrophy,” was published in the journal EMBO…

Following its failure to increase dystrophin levels of patients in an early-stage clinical trial, Wave Life Sciences has discontinued development of the investigational exon-skipping therapy suvodirsen for people with Duchenne muscular dystrophy (DMD). Lack of dystrophin, an essential protein for healthy muscles, causes DMD. Patients may…

A new technique that compares RNA production between an individual’s two gene copies may help find genetic alterations that lead to muscular dystrophy and other rare diseases, a study suggests. The research, “Genetic regulatory variation in populations informs transcriptome analysis in rare disease,” appeared in the…

Owing to a serious adverse event in a patient, the U.S. Food and Drug Administration (FDA) has put another clinical hold on a Phase 1/2 clinical trial of SGT-001, an investigational gene therapy for Duchenne muscular dystrophy (DMD). Solid Biosciences, the company developing SGT-001, is working…

A novel one-time gene therapy that leads to the production of a dystrophin-related protein called utrophin prevents muscle deterioration and results in muscle regeneration in animal models of Duchenne muscular dystrophy (DMD) without triggering an immune response, a new study reports. The study, “Non-immunogenic…