The U.S. Food and Drug Administration recently granted rare pediatric disease designation to Myonexus Therapeutics’ experimental gene therapy MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E, or LGMD2E. The announcement was made by Myonexus and Nationwide Children’s Hospital. The designation allows priority review for the…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to investigational therapy Sarconeos for Duchenne muscular dystrophy (DMD), Biophytis, the therapy’s developer, announced. The company also has filed a similar application with the European Medicines Agency (EMA). A decision is is expected…
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
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…
Whether you or your loved one has a chronic illness, it’s important to prepare for hospital stays. Despite my many visits to the emergency room, I’ve only had to stay in the hospital a few times. We keep a suitcase in the car full of essentials for an…
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’Â 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…
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