Publications

Primary Hyperoxaluria Type 1 (PH1)

Quantification of oxalate by novel LC-MS/MS: assay development, validation and application in lumasiran clinical trials

Bioanalysis

Author(s)

Valerie A Clausen, Karen H Cao, John M Gansner, et al

Acute Hepatic Porphyria (AHP)

Pharmacokinetic-Pharmacodynamic Model of Urinary δ-Aminolevulinic Acid Reduction After Givosiran Treatment in Patients with Acute Hepatic Porphyria

CPT: Pharmacometrics & Systems Pharmacology

Author(s)

Jongtae Lee, Megan Melch, Gabriel J Robbie

Primary Hyperoxaluria Type 1 (PH1)

Estimating health state utilities in primary hyperoxaluria type 1: a valuation study

Journal of Medical Economics

Author(s)

Hayley M de Freitas, David Danese, Lena Hubig, et al

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