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The Muscular Dystrophy Association (Â鶹Éç) has awarded Locana $550,000 through its Â鶹Éç Venture Philanthropy (MVP) program to help advance the biotech company’s therapy platform for myotonic dystrophy (DM). Specifically, the grant to the San Diego, California-based gene therapy company is aimed at furthering the development of its RNA-targeting…

It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…

Increasing the amount of a protein called sarcospan may help to protect against heart failure in children with Duchenne muscular dystrophy (Â鶹Éç), a study in mice suggests. The study, “Stabilization of the cardiac sarcolemma by sarcospan rescues Â鶹Éç-associated cardiomyopathy,” was published in the journal JCI Insight. Despite significant advances…

Enrollment is progressing quickly in Catabasis Pharmaceuticals‘ pivotal Phase 3 PolarisÂ鶹Éç trial, testing its investigational small molecule therapy edasalonexent for Duchenne muscular dystrophy (Â鶹Éç) — with sites in some countries already full, the company said. Screening is ongoing in the U.S., Canada, Australia, the U.K.,…

Catabasis Pharmaceuticals announced new preclinical data showing that, in a mouse model of Duchenne muscular dystrophy (Â鶹Éç), the small molecule edasalonexent preserved bone health, whereas the corticosteroid prednisolone caused bone loss. An ongoing double-blind and randomized Phase 3 trial (NCT03703882), called PolarisÂ鶹Éç, is evaluating…

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…

The European Medicines Agency (EMA) has validated a marketing authorization application for Puldysa (idebenone) for treating respiratory dysfunction in Duchenne muscular dystrophy (Â鶹Éç) patients who do not take glucocorticoids. This means that the submission, which was made as a conditional marketing authorization (potentially granted to therapies whose immediate benefit…

It’s been 15 years since Sailormen, a Popeyes Louisiana Kitchen franchise, first partnered with the Muscular Dystrophy Association (Â鶹Éç) to enhance the lives of patients. It’s keeping at it, netting $681,015 in its latest efforts. The money was raised at the 15th Annual Sailormen Â鶹Éç Golf Classic and the…

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…

Biophytis SA is collaborating with AFM-Telethon to advance an oral pediatric formulation of Sarconeos (BIO101) for treating Duchenne muscular dystrophy (Â鶹Éç). The pact calls for the French muscular dystrophy organization AFM-Telethon to give €400,000 (about $452,000) to France-based Biophytis  for further preclinical study. The funds also will go…