Exondys 51 (eteplirsen), an RNA-targeted therapy marketed by Sarepta Therapeutics, is the first approved treatment targeting the underlying cause of Duchenne muscular dystrophy (DMD). Sarepta is continuing to refine its RNA-targeted therapies, with a goal of being able to transport more of the treatment…
Celebrex (celecoxib), an FDA-approved anti-inflammatory and pain reliever marketed by Pfizer, showed an ability to improve strength and function in leg, heart and diaphragm muscles of an animal model of Duchenne muscular dystrophy (DMD), a study found. Mice treated daily for one month with celecoxib also showed increases in…
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…
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