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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.
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Primary Hyperoxaluria Type 1 (PH1)

Qualitative assessment of the patient experience of primary hyperoxaluria type 1: an observational study

BMC Nephrology

Author(s)

David Danese, Diana Goss, Carla Romano, et al

October 2023

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Transthyretin Amyloidosis (ATTR)

Pharmacokinetics and Pharmacodynamics of Patisiran in Patients with hATTR Amyloidosis and with Polyneuropathy After Liver Transplantation

Clinical Pharmacokinetics

Author(s)

Prajakta Badri, Bahru Habtemariam, Megan Melch, et al

August 2023

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Transthyretin Amyloidosis (ATTR)

Treatment response and neurofilament light chain levels with long-term patisiran in hereditary transthyretin-mediated amyloidosis with polyneuropathy: 24-month results of an open-label extension study

Amyloid

Author(s)

Simina Ticau, Emre Aldinc, Michael Polydefkis, et al

July 2023

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Transthyretin Amyloidosis (ATTR)

Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy

Neurology and Therapy

Author(s)

Laura Obici, Senda Ajroud-Driss, Kon-Ping Lin, et al

July 2023

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Cardiovascular (CV)

Zilebesiran, an RNA Interference Therapeutic Agent for Hypertension

New England Journal of Medicine

Author(s)

Akshay Desai, David Webb, Jorg Taubel, et al

July 2023

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Acute Hepatic Porphyria (AHP)

Efficacy and safety of givosiran for acute hepatic porphyria: Final results of the randomized phase III ENVISION trial

Journal of Hepatology

Author(s)

David J Kuter, Herbert L Bonkovsky, Susana Monroy, et al

July 2023

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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

May 2023

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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

May 2023

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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

March 2023

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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

March 2023

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