Publications

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

Transthyretin Amyloidosis (ATTR)

Presence of the V122I Variant of Hereditary Transthyretin-Mediated Amyloidosis Among Self-Reported White Individuals in a Sponsored Genetic Testing Program

Circulation: Genomic and Precision Medicine

Author(s)

Barry Trachtenberg, Sachin Shah, Robert Nussbaum, et al

Primary Hyperoxaluria Type 1 (PH1)

Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial

Clinical Journal of the American Society of Nephrology

Author(s)

Yaacov Frishberg, Georges Deschênes, Jaap W Groothoff, et al

Primary Hyperoxaluria Type 1 (PH1)

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

New England Journal of Medicine

Author(s)

Sander F Garrelfs, Yaacov Frishberg, Sally A Hulton, et al