Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study.
Bruce WangPaolo VenturaKei-Ichiro TakaseManish ThaparDavid CassimanIlja KubischShangbin LiuMarianne T SweetserManisha BalwaniPublished in: Orphanet journal of rare diseases (2022)
Patients with AHP, regardless of annualized attack rates, have considerable disease burden that may partly be alleviated with givosiran.
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