Givosiran for the treatment of acute hepatic porphyria.

Published in: Expert review of clinical pharmacology (2022)

Givosiran is a small interfering RNA (siRNA) therapeutic that reduces hepatic activity of ALAS1 and decreases accumulation of neurotoxic porphyrin precursors in patients with AHPs, ultimately reducing the number of acute attacks and improving symptoms and QoL between attacks. As AHPs are lifelong diseases, long-term safety data are needed for givosiran as an siRNA-based therapy.

Keyphrases

liver failure
respiratory failure
drug induced
cancer therapy
aortic dissection
electronic health record
hepatitis b virus
big data
hyaluronic acid
combination therapy
physical activity