News UCB bags first EU approval for rare genetic disease TK2d UCB gets an EU recommendation for the first therapy for thymidine kinase 2 deficiency (TK2d), plus other CHMP decisions.
News UCB bags FDA okay for first drug to treat rare disease TK2d People living with thymidine kinase 2 deficiency (TK2d), a vanishingly rare and often fatal genetic disorder, finally have an approved therapy.
News FDA knocks back Pharming's bid for wider Joenja use The FDA has turned down Pharming's bid to broaden the label of Joenja for rare immunodeficiency disorder APDS so it can be used in younger patients.
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