Members of the Heiltsuk Nation in Bella Bella, B.C., are celebrating a joyful arrival with the…
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Advancing diagnostics reagent development for emerging and rare diseases
The World Health Organization (WHO) has drafted a priority pathogen list to direct funding and resources…
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Mom says infant may have rare Powassan virus tick-borne disease
A Massachusetts mom says her infant daughter may be the patient that state health officials recently…
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Bridging psychiatry and rare genetic diseases: a scoping review of therapeutic strategies and diagnostic delay paired with healthcare economic burden analysis | Orphanet Journal of Rare Diseases
Nguengang Wakap S, Lambert DM, Olry A, Rodwell C, Gueydan C, Lanneau V, Murphy D, Le…
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Baby with rare disease given world-first personal CRISPR gene therapy
Baby KJ after a gene-editing infusion with researchers Rebecca Ahrens-Nicklas and Kiran Musunuru Children’s Hospital of…
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Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment
News Release Thursday, May 15, 2025 NIH-supported gene-editing platform lays groundwork to rapidly develop treatments…
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