Publications

Acute Hepatic Porphyria (AHP)

Population Pharmacokinetic Analysis of the RNAi Therapeutic Givosiran in Patients with Acute Hepatic Porphyria

Clinical Pharmacokinetics

Author(s)

Megan Melch, Jongtae Lee, Claudia Jomphe, et al

Transthyretin Amyloidosis (ATTR)

Effect of patisiran on stroke volume in hereditary transthyretin-mediated amyloidosis: insights from pressure–volume analysis of the APOLLO study

European Journal of Heart Failure

Author(s)

Hannah Rosenblum, Jan Griffin, Masatoshi Minamisawa, et al

Transthyretin Amyloidosis (ATTR)

Impact of baseline polyneuropathy severity on patisiran treatment outcomes in the APOLLO trial

Amyloid

Author(s)

Dianna Quan, Laura Obici, John Ber, et al

Acute Hepatic Porphyria (AHP)

EXPLORE B: A prospective, long-term natural history study of patients with acute hepatic porphyria with chronic symptoms

Journal of Inherited Metabolic Disease

Author(s)

David Cassiman, Raili Kauppinen, Susana Monroy

Transthyretin Amyloidosis (ATTR)

Gene Silencing Therapeutics in Cardiology: A Review Article

International Journal of Cardiovascular Sciences

Author(s)

Patrick Jay, Martin Maier, Laura Saltonstall, et al

Primary Hyperoxaluria Type 1 (PH1)

Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial

Pediatric Nephrology

Author(s)

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

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