Publications

Acute Hepatic Porphyria (AHP)

Hyperhomocysteinemia in acute hepatic porphyria (AHP) and implications for treatment with givosiran

Expert Review of Gastroenterology & Hepatology

Author(s)

Paolo Ventura, Eliane Sardh, Nicola Longo, et al

Acute Hepatic Porphyria (AHP)

Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study

Orphanet Journal of Rare Diseases

Author(s)

Bruce Wang, Paolo Ventura, Kei-Ichiro Takase, et al

Transthyretin Amyloidosis (ATTR)

Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial

Amyloid

Author(s)

David Adams, Ivailo Tournev, Mark Taylor, et al

Primary Hyperoxaluria Type 1 (PH1)

Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial

American Journal of Kidney Diseases

Author(s)

Mini Michael, Jaap W Grothoff, Hadas Shasha-Lavsky, et al

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

Author(s)

Kristen Wheeden, Desiree Lyon Howe, Sue Burrell, et al

Transthyretin Amyloidosis (ATTR)

Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation

American Journal of Transplantation

Author(s)

Hartmut Schmidt, Jonas Wixner, Violaine Planté-Bordeneuve, et al

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

Author(s)

Sally A Hulton, Jaap W Groothoff, Yaacov Frishberg, et al

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

Author(s)

David J Sas, Daniella Magen, Wesley Hayes, et al

Primary Hyperoxaluria Type 1 (PH1)

Primary Hyperoxaluria Type 1 Disease Manifestations and Healthcare Utilization: A Multi-Country, Online, Chart Review Study

Frontiers in Medicine

Author(s)

Xiangling Wang, David Danese, Thomas Brown, et al

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

Author(s)

Daphne Vassiliou, Eliane Sardh, Pauline Harper, et al