News

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…

Atamyo Therapeutics is seeking approvals in Europe to launch its first trial testing ATA-100, its investigational gene therapy for a type of limb-girdle muscular dystrophy (LGMD). The company, a spin-off from Genethon, a Paris-based genetics research laboratory, has submitted an application asking three European…

The Newborn Screening Pilot program run in New York by Parent Project Muscular Dystrophy (PMDD) is now complete, having screened more than 36,000 babies across the state for a muscular dystrophy over the last two years. Significantly high levels of creatine kinase, an enzyme released by damaged muscle cells that can indicate…

Flying can be an ordeal for many people with disabilities who rely on wheelchairs to get from point A to point B. Sometimes disabled travelers get to their destination only to realize their wheelchair or scooter is broken or missing. In 2019, the year after airlines were required to release…

The COVID-19 pandemic has had a substantial impact on the mental health and quality of life of people affected by muscular dystrophy (MD). One big underlying issue for the families of those with Duchenne and Becker muscular dystrophies is that the pandemic has caused many patients to…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented communities of color. “For rare disease patients, there are many challenges — and for people of color with a rare disease, these challenges are compounded…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to AOC 1001, an investigational treatment for myotonic dystrophy type 1, known as DM1, a late-developing muscle disorder. The designation will enable AOC 1001’s developer, Avidity Biosciences, to have more frequent interactions with the FDA throughout…

New data across clinical trials of SRP-9001 show the investigative gene therapy induces sustained functional improvements in people with Duchenne muscular dystrophy (DMD), and does so with good tolerability, Sarepta Therapeutics, its developer, announced. The company, in presenting these data, also offered more details about the recently launched and pivotal…

The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…

Tamoxifen, commonly used to treat breast cancer, failed to meet its primary goal — delaying disease progression — in a Phase 3 study evaluating its use in boys with Duchenne muscular dystrophy (DMD), preliminary data show. Boys treated with tamoxifen and those given a placebo over 48 weeks in the…