Acute Hepatic Porphyria (AHP)
Hyperhomocysteinemia in acute hepatic porphyria (AHP) and implications for treatment with givosiran
Expert Review of Gastroenterology & Hepatology
Acute Hepatic Porphyria (AHP)
Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study
Orphanet Journal of Rare Diseases
Transthyretin Amyloidosis (ATTR)
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial
Amyloid
Primary Hyperoxaluria Type 1 (PH1)
Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial
American Journal of Kidney Diseases
Acute Hepatic Porphyria (AHP)
Patient Perspective on Acute Hepatic Porphyria with Sporadic Attacks: A Chronic Disease with Substantial Health-Related Quality of Life Impacts
Advances in Therapy
Transthyretin Amyloidosis (ATTR)
Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation
American Journal of Transplantation
Primary Hyperoxaluria Type 1 (PH1)
Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1
Kidney International Reports
Primary Hyperoxaluria Type 1 (PH1)
Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children
Genetics in Medicine
Primary Hyperoxaluria Type 1 (PH1)
Primary Hyperoxaluria Type 1 Disease Manifestations and Healthcare Utilization: A Multi-Country, Online, Chart Review Study
Frontiers in Medicine
Acute Hepatic Porphyria (AHP)
A Drug-Drug Interaction Study Evaluating the Effect of Givosiran, a Small Interfering Ribonucleic Acid, on Cytochrome P450 Activity in the Liver
Clinical Pharmacology and Therapeutics
