Wave Life Sciences’ treatment candidate WVE-210201 has been granted both orphan drug designation and rare pediatric disease designation for the treatment of Duchenne muscular dystrophy (DMD) from the U.S. Food and Drug Administration. Orphan drug designation provides Wave with seven years of market exclusivity in the U.S., tax…
Jiffy Lube is teaming up with the Muscular Dystrophy Association (MDA) in the annual MUSCLE UP! campaign for the seventh year in a row. The campaign, which runs through the month of August, aims to help families affected by muscular dystrophy and other muscle-debilitating diseases. Customers donating $3 or…
Two small patient advocacy groups, Little Hercules Foundation and Team Joseph, are stepping up their joint Duchenne Family Assistance Program (DFAP). The expanded program, created last October, comes in response to the increasing rate of insurance company denials of life-extending treatments and medical devices for Duchenne muscular dystrophy patients.
The first patient has been enrolled in a Phase 2 trial evaluating the effectiveness and safety of MNK-1411, an investigational therapy for Duchenne muscular dystrophy (DMD), announced Mallinckrodt Pharmaceuticals, the treatment’s developer. The company also reported that MNK-1411 was…
Duchenne muscular dystrophy affects an estimated 1 in 3,500 male births. But Elizabeth Heller is an outlier even among her rare friends. The Chicago-born Heller, who relocated to Tempe, Arizona, three years ago, is one of only a handful of women with Duchenne in the United States. According to the…
Nicholas Johnson, vice chair of research and associate professor of neurology at Virginia Commonwealth University in Richmond, was awarded a $700,000 grant from the Muscular Dystrophy Association (MDA) to establish the Limb-Girdle Muscular Dystrophy (LGMD) Clinical Research Network…
The nonprofit group CureDuchenne and Connecticut-based rareLife Solutions have launched DuchenneXchange — a dedicated online platform where those interested in Duchenne muscular dystrophy “can share comprehensive, safe, credible resources and information, as well as get the support they need from diagnosis to treatment to clinical trials.” The project, announced in a …
Gavin Ward, a spunky 8-year-old Arkansas boy with Duchenne, enjoys building contraptions with his Lego set and watching video games. He also walks up to 18,000 steps a day and accompanies his father, Bruce, on outings near their home in Royal, about 70 miles west of Little Rock. But what…
A Phase 1/2 trial evaluating Sarepta’s microdystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD) was placed on clinical hold by the U.S. Food and Drug Administration. The FDA found trace amounts of plasmid DNA…
Insilico Medicine and A2A Pharmaceuticals, two leading artificial intelligence (AI) biotechs, are collaborating to create a new company called Consortium.AI, which will advance AI for the development of new molecules to treat Duchenne muscular dystrophy (DMD) and other orphan diseases. Consortium.AI will feature computational new candidates designed…
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