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The gene therapy EtranaDez for hemophilia B placed on clinical hold after a patient was diagnosed with liver cancer, but UniQure points to that patient’s extensive history of liver disease.

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BioCryst’s Oral Option To Take On Injectables In HAE Prophylaxis Market

Orladeyo is the first oral prophylactic approved in the US for hereditary angioedema, with Japan and EU approvals expected to follow. Pricing of $485,000 a year undercuts segment leader.

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