Acute Intermittent Porphyria: An Overview of Therapy Developments and Future Perspectives Focusing on Stabilisation of HMBS and Proteostasis Regulators.
Bustad HJ, Kallio JP, Vorland M, Fiorentino V, Sandberg S, Schmitt C, Aarsand AK, Martinez A.
Int J Mol Sci. 2021 Jan 12;22(2):675. doi: 10.3390/ijms22020675.
PMID:33445488
Givosiran for Acute Intermittent Porphyria.
Gomá-Garcés E, Pérez-Gómez MV, Ortíz A.
N Engl J Med. 2020 Nov 12;383(20):1989. doi: 10.1056/NEJMc2026458.
PMID:33176091
Acute intermittent porphyria: Acute flare-up resolved with givosiran.