Renal Function Decline With Small Interfering RNA Silencing Aminolevulinic Acid Synthase 1 (ALAS1)

Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid (siRNA), Givosiran, in Patients with Acute Hepatic Porphyria

(2020) Sagar Agarwal et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

(2020) Manisha Balwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

(2019) Eliane Sardh et al.   NEW ENGLAND JOURNAL OF MEDICINE

Porphyria

(2017) D. Montgomery Bissell et al.   NEW ENGLAND JOURNAL OF MEDICINE

High prevalence of and potential mechanisms for chronic kidney disease in patients with acute intermittent porphyria

(2015) Nicolas Pallet et al.   KIDNEY INTERNATIONAL

RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice

(2014) M. Yasuda et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Establishing a network of specialist Porphyria centres - effects on diagnostic activities and services

(2012) Mette C Tollånes et al.   Orphanet Journal of Rare Diseases

Porphyrias

(2010) Hervé Puy et al.   LANCET