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Trending in Rare Diseases: FDA Approvals, New Therapies and More
Автор: LucidQuest
Загружено: 2025-12-21
Просмотров: 3
Описание: Novartis has received FDA approval for Ianalumab, offering extended disease control in immune thrombocytopenia with just four monthly doses. The BioIndustry Association calls for faster access to rare disease treatments in the UK. NORD urges Congress to reauthorize the Rare Pediatric Disease Voucher Program. The FDA approves Waskyra, the first gene therapy for Wiskott-Aldrich Syndrome, while Amgen's UPLIZNA® gains approval for generalized myasthenia gravis. UCB’s GEMZ study shows promising results for CDKL5 deficiency disorder, and the Kennedy administration adds DMD and MLD to newborn screening.
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