FDA approves first treatment for inherited rare disease
Express Pharma|December 01-15, 2019
The approval of the drug, Givlaari was based on the results of a clinical trial of 94 patients with acute hepatic porphyria
FDA approves first treatment for inherited rare disease

THE US Food and DrugAdministration grantedapproval to Givlaari (givosiran) for the treatment of adult patients with acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme (which helps bind oxygen in the blood).

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