News Italy leads the way on child genetic disease diagnosis A scheme to hasten the diagnosis of children with genetic disorders is achieving strong results and could be a template for national health systems.
News FDA clears first gene therapy for rare genetic disease WAS Fondazione Telethon's Waskyra has become the first gene therapy in the US for Wiskott-Aldrich syndrome, and the first taken to market by a non-profit.
News FDA clears first drug for MCT8 deficiency Sweden's Egetis has won approval in the US for the first-ever treatment for people living with the ultra-rare genetic disease MCT8 deficiency.
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