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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)

Impact of Baseline Neuropathy Severity on Vutrisiran Treatment Response in the Phase 3 HELIOS-A Study

Neurology and Therapy

Author(s)

Marco Luigetti, Dianna Quan, John L. Berk, et al

Acute Hepatic Porphyria (AHP)

Elevated homocysteine is negatively correlated with plasma cystathionine β ‐synthase activity in givosiran‐treated patients

Journal of Inherited Metabolic Disease Reports

Author(s)

Mark A. Keibler, Gautham V. Sridharan, Marianne T. Sweetser, et al

Transthyretin Amyloidosis (ATTR)

Patisiran, an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis: Sub-analysis in Taiwanese patients from the APOLLO study

Journal of the Formosan Medical Association

Author(s)

Lin KP, Yang CC, Lee YC, et al

Cardiovascular (CV)

RNA Interference With Zilebesiran for Mild to Moderate Hypertension: The KARDIA-1 Randomized Clinical Trial

The Journal of the American Medical Association

Author(s)

George L Bakris, Manish Saxena, Anil Gupta, et al

Transthyretin Amyloidosis (ATTR)

Impact of vutrisiran on exploratory cardiac parameters in hereditary transthyretin-mediated amyloidosis with polyneuropathy

European Journal of Heart Failure

Author(s)

Pablo Garcia-Pavia, Martha Grogan, Parag Kale, et al

Primary Hyperoxaluria Type 1 (PH1)

Distinguishing characteristics of pediatric patients with primary hyperoxaluria type 1 in PEDSnet

Journal of Pediatric Urology

Author(s)

Gregory E. Tasian, Kimberley Dickinson, Grace Park, et al

Acute Hepatic Porphyria (AHP)

Clinical features of acute attacks, chronic symptoms, and long-term complications among patients with acute hepatic porphyria in Japan: a real-world claims database study

Orphanet Journal of Rare Diseases

Author(s)

Yutaka Horie, Yuka Yasuoka & Tomohide Adachi

Transthyretin Amyloidosis (ATTR)

Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis

New England Journal of Medicine

Author(s)

Mathew Maurer, Parag Kale, Marianna Fontana, et al

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

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