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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 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

Primary Hyperoxaluria Type 1 (PH1)

Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial

American Journal of Kidney Diseases

Author(s)

Mini Michael, Jaap W Grothoff, Hadas Shasha-Lavsky, et al

Acute Hepatic Porphyria (AHP)

Patient Perspective on Acute Hepatic Porphyria with Sporadic Attacks: A Chronic Disease with Substantial Health-Related Quality of Life Impacts

Advances in Therapy

Author(s)

Kristen Wheeden, Desiree Lyon Howe, Sue Burrell, et al