Latest News
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PARIS, France – Professor Francesco Muntoni, principal investigator of the international multicenter gene therapy trial for Duchenne Muscular Dystrophy with GNT0004 product, sponsored by Genethon, presented the innovative trial design and its initial results at the Myology 2024 international scientific congress, currently taking place in Paris. The trial combines phases...
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BOSTON, Mass. — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the publication of results from a Fabry disease patient survey in the peer-reviewed Orphanet Journal of Rare Diseases. “There continues to...
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BOULDER, Colo. — Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designations for sevasemten for the treatment of Becker muscular dystrophy (Becker) and for the treatment of Duchenne muscular dystrophy (Duchenne). Sevasemten is an investigational...