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…
Either by coincidence or by design, MDA Hellas, the Muscular Dystrophy Association of Greece, is located on Elpidos Street in downtown Athens. Elpidos is Greek for “hope” — and that’s exactly what MDA Hellas offers the many neuromuscular disease patients under its care. Antigone Karras is executive manager of…
In the mythology of ancient Greece, Hermes was the god of boundaries and transitions, with the ability to move between the mortal and divine worlds. In modern-day Athens, Hermes Christos Athanasiou Gill is a cheerful, trilingual 7-year-old who likes math and enjoys playing Minecraft, a video game. He also has…
Treatment of Duchenne muscular dystrophy (DMD) patients with Raxone (idebenone) delays the need for assisted ventilation by three years, new data from a Phase 3 clinical trial show. Trial results also demonstrate that measuring the air flow speed in and out of the lungs, or peak expiratory flow…
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