News

The U.S. Food and Drug Administration has granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping Duchenne muscular dystrophy (DMD) treatment from NS Pharma. Treatments granted this designation are aimed at serious or life-threatening diseases affecting children and fewer than 200,000 U.S. patients. “We are grateful for this…

Atamyo Therapeutics has partnered with the Dion Foundation for Children with Rare Diseases to expand into the U.S. a clinical trial of ATA-200, a potential gene therapy for limb-girdle muscular dystrophy (LGMD) type 2C/R5 — dubbed LGMD2C/R5. The Phase 1b study (NCT05973630), designed to evaluate the…

Up to a year of treatment with DYNE-251, Dyne Therapeutics’ investigational exon 51-skipping therapy, led to improvements in motor function for boys with Duchenne muscular dystrophy (DMD), according to a clinical trial update. Based on these positive data from the Phase 1/2 DELIVER clinical trial (NCT05524883), Dyne…

A noninvasive quantitative MRI, or qMRI, was found to detect early muscle abnormalities among people with limb-girdle muscular dystrophy type R1 (LGMDR1), according to a small study from Europe. Many of the qMRI findings correlated with clinical assessments of muscle function and patient-reported activities. “Our findings revealed alterations in…

The muscular dystrophy (MD) community is poised to mark National Muscular Dystrophy Awareness Month with events throughout September to call attention to MD and related neuromuscular disorders — and to raise critical funds for the more than 300,000 U.S. families thought to be affected. The annual observance was…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to RAG-18, being developed as a potential treatment for both Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). RAG-18 is a small activating RNA (saRNA) therapy from Ractigen Therapeutics that’s designed to counteract the shortage…

A little over a year ago, neurologist Sarah Wright administered Elevidys (delandistrogene moxeparvovec-rokl) to then 5-year-old Hiram Secrist, making him the first Duchenne muscular dystrophy (DMD) patient to receive the gene therapy outside of a clinical trial. Elevidys became the first and only gene therapy available for DMD…

The U.S. Food and Drug Administration (FDA) has given WVE-N531, an investigational therapy for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping, a rare pediatric drug designation. This status aims to incentivize companies developing treatments for serious or life-threatening conditions that primarily affect children and are considered…

Avidity Biosciences‘ delpacibart zotadirsen, or del-zota for short — formerly called AOC 1044 — was found to safely increase levels of dystrophin production to 25% of normal in people with Duchenne muscular dystrophy (DMD), according to new data from the EXPLORE44 trial. In the Phase 1/2 clinical…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD) that ultimately aims to slow the progression of the genetic condition. The FDA awards this status to investigational medicines that are designed to treat serious,…