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 Sanofi's type 1 diabetes drug poised for EU rollout Sanofi is celebrating an approval recommendation in the EU for Teizeild, a first-in-class drug designed to delay the progression of type 1 diabetes.
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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