News

The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in its 28 member countries, officials meeting last week in Vienna said. More than 900 people from 58 nations attended the 9th European Conference on Rare Diseases & Orphan Drugs (ECRD),…

Sarepta Therapeutics has established an exclusive collaboration agreement with Myonexus Therapeutics to develop five new therapies targeting various forms of limb-girdle muscular dystrophy (LGMD). These include three clinical and two preclinical stage programs for the most severe and common forms of LGMD. With this partnership, Sarepta will expand…

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…