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Publications

Discover peer-reviewed publications by therapeutic area.
The information on this page pertains to select publication resources. It is not intended to serve as a comprehensive list of all publications resources.
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Any content about investigational therapeutics or investigational uses of locally approved products does not establish the safety or efficacy of these therapeutics or uses, and there is no guarantee of local regulatory approval.
The provision of this content is not intended to recommend or suggest use of its products in any manner inconsistent with local regulatory approval. For questions beyond a product’s authorized indications or for information not available here, please contact Alnylam Medical Information.

Publications

Transthyretin Amyloidosis (ATTR)

Indirect treatment comparison (ITC) of the efficacy of vutrisiran and tafamidis for hereditary transthyretin-mediated amyloidosis with polyneuropathy

Expert Opinion on Pharmacotherapy

Author(s)

Madeline Merkel, David Danese, Chongshu Chen, et al

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