Givosiran per al tractament de la porfíria hepàtica aguda
, 2022 Les porfíries són un grup de malalties metabòliques causades per una pèrdua de funcionalitat en els enzims que participen en la biosíntesi del grup hemo. Es classifiquen en funció de les principals manifestacions clíniques (neurològiques o cutànies) i del teixit on es produeix l’alteració enzimàtica (fetge o medul·la òssia).
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