Andrea Lobo,  —

The European Commission (EC) has granted orphan drug designation to AB-1003, an investigational gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9), being developed by Asklepios BioPharmaceutical (AskBio). This designation is given to therapies for life-threatening or chronically debilitating diseases — those affecting no more than five…

GIVI-MPC, a stem cell therapy to create new muscle in Duchenne muscular dystrophy (DMD), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status encourages the development of therapies for rare diseases — those affecting fewer than 200,000 people in the U.S.

Whole-body muscle MRI correlates with functional measures in children with facioscapulohumeral muscular dystrophy (FSHD) and might help evaluate disease severity, according to a study. “This study demonstrates that MR muscle imaging is a potential biomarker of disease staging in pediatric FSHD,” the researchers wrote in “Correlation between whole body…

The first patients have consented to share their clinical data with the Electronic Health Record (EHR) Study, according to an announcement from Parent Project Muscular Dystrophy (PPMD). The study’s goal is to help clinicians and researchers improve care and learn more about Duchenne and Becker muscular dystrophy. The…

Blocking a calcium channel called TRPC6 prolonged survival in a mouse model of Duchenne muscular dystrophy (DMD) by improving skeletal and heart muscle function and reducing bone deformities, a study showed. Its results support clinical trials of TRPC6 inhibitors in Duchenne patients as “a novel DMD therapy,” the researchers wrote,…

The first patient has received Dyne Therapeutics‘ investigational therapy DYNE-251 for treating Duchenne muscular dystrophy (DMD), the company has announced. The therapy is being tested in the DELIVER Phase 1/2 clinical trial and is indicated for patients with mutations amenable to exon 51 skipping. “We are excited to…