Stay updated with the latest developments in the field of Muscle Dystrophy through these recent news articles
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the House of Representatives’ Energy and Commerce Health Subcommittee voted to advance several bipartisan bills important to the rare disease community, two of which encourage rare disease drug development and two that would make it easier for many rare disease patients to access necessary care.
Regulators in France and Italy have given Atamyo Therapeutics the green light to launch a clinical trial testing ATA-200, an investigational gene therapy for children with a type of limb-girdle muscular dystrophy called LGMD2C or LGMDR5.
Genethon announced today the first positive clinical results obtained with the on-going ATA-001 Phase 1b/2b gene therapy clinical trial for the treatment of fukutin-related protein (FKRP) limb-girdle muscular dystrophy (LGMD2I/R9).
