News

The Duchenne Registry, formerly known as DuchenneConnect, marks its 10th anniversary as the world’s largest repository of patient-reported data on Duchenne muscular dystrophy (DMD): 4,200 people from more than 100 countries with both DMD and Becker muscular dystrophy. A member of the Treat NMD-International Neuromuscular Network, the registry began…

Capricor Therapeutics’ Phase 2 clinical trial to evaluate the safety and effectiveness of CAP-1002 in boys and young men with Duchenne muscular dystrophy (DMD) has started recruiting participants. The HOPE-2 trial (NCT03406780) is planned to enroll up to 84 participants ages 10 or older with advanced stages…

Duchene muscular dystrophy (DMD) patients now have a new scholarship program that could help them pursue their post-high school education goals. Sarepta Therapeutics recently launched “Route 79, the Duchenne Scholarship Program” to help up to 10 students who are taking the lead and making educational choices to…

The U.S. Food and Drug Administration (FDA) recently granted fast track designation to Acceleron Pharma’s ACE-083 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). ACE-083 is a locally-acting agent that binds to and inhibits proteins in the transforming growth factor (TGF)-beta family, such as myostatin, which reduce…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

The investigational therapy edasalonexent was seen to slow disease progression in boys with Duchenne muscular dystrophy, almost one-year results from the MoveDMD trial report. Specifically, edasalonexent — administered orally as 100 mg/kg dose for 48 weeks — resulted in a statistically significant delay in disease progression and signs of…

The U.S. Food and Drug Administration (FDA) granted orphan drug status to MYO-101, a gene therapy being developed by Myonexus Therapeutics for the treatment of limb girdle muscular dystrophy type 2E (LGMD2E) . The FDA’s orphan drug designation is recognition of the potential of MYO-101 for the treatment of…

A national survey to identify how Americans with disabilities are coping with work and overcoming barriers to employment has found that, despite persistent challenges, their efforts are making a big difference. The 2015 Kessler Foundation National Employment and Disability Survey (KFNEDS) was conducted by a multidisciplinary team of researchers at…

It’s not every day a Southeast Asian monarchy bestows a $100,000 prize on a U.S. government research agency. In fact, it rarely happens. But this year is different. Thailand’s Prince Mahidol Award Foundation has chosen the National Human Genome Research Institute — a division of the National Institutes of Health (NIH)…

A potential next-generation therapy, golodirsen (SRP-4053) facilitates and increases dystrophin production in Duchenne muscular dystrophy (DMD) patients, an interim analysis of a Phase 1/2 trial being presented at the 2018 American Academy of Neurology (AAN) Annual Meeting in Los Angeles reports. These findings, in  “Golodirsen Induces Exon Skipping Leading…