Porphyrias are a group of metabolic diseases caused by a loss of functionality in the enzymes involved in the biosynthesis of the heme group. They are classified according to the main clinical manifestations (neurological or cutaneous) and the tissue where the enzymatic alteration occurs (liver or bone marrow). Thus, those that affect the liver are called hepatic porphyria
Givosiran; Acute hepatic porphyria; Minority disease
Programa d'Harmonització Farmacoterapèutica. Givosiran per al tractament de la porfíria hepàtica aguda. Barcelona: Servei Català de la Salut; 2021.
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