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.
Acute Hepatic Porphyria (AHP)
Detecting rare diseases in electronic health records using machine learning and knowledge engineering: Case study of acute hepatic porphyria
PLOS One
Acute Hepatic Porphyria (AHP)
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria
New England Journal of Medicine
Transthyretin Amyloidosis (ATTR)
DISCOVERY: prevalence of transthyretin (TTR) mutations in a US-centric patient population suspected of having cardiac amyloidosis
Amyloid
Cardiovascular (CV)
RNA interference therapeutics targeting angiotensinogen ameliorate preeclamptic phenotype in rodent models
The Journal of Clinical Investigation
Primary Hyperoxaluria Type 1 (PH1)
Characterising a healthy adult with a rare HAO1 knockout to support a therapeutic strategy for primary hyperoxaluria
eLife
Acute Hepatic Porphyria (AHP)
Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid, Givosiran, in Patients With Acute Hepatic Porphyria
Clinical Pharmacology and Therapeutics
Acute Hepatic Porphyria (AHP)
EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks
Hepatology
Transthyretin Amyloidosis (ATTR)
Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure-Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis
Journal of Clinical Pharmacology
Cardiovascular (CV)
Strong and Sustained Antihypertensive Effect of Small Interfering RNA Targeting Liver Angiotensinogen
Hypertension
Transthyretin Amyloidosis (ATTR)
Association of Patisiran, an RNA Interference Therapeutic, With Regional Left Ventricular Myocardial Strain in Hereditary Transthyretin Amyloidosis: The APOLLO Study
JAMA Cardiology
