Search Results for "RNAi"

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Amyloid-related imaging abnormalities in an ongoing Phase 1 study of mivelsiran, an investigational RNA interference therapeutic targeting amyloid precursor protein, in patients with Alzheimer’s disease

APOLLO Post-Hoc Analysis by Baseline Polyneuropathy Severity

A post-hoc analysis of the APOLLO trial was performed to evaluate the use of patisiran compared to placebo by baseline NIS quartile in patients with the the polyneuropathy of hATTR. This infographic summarizes the post-hoc analysis results, which were published in the journal Amyloid.

Impact of Vutrisiran on Activities of Daily Living and Functional Status in Patients with hATTR Amyloidosis

Long-term treatment with lumasiran: final results from the phase 2 open-label extension study

Patient Experience with Acute Hepatic Porphyria Before and After Long-Term Givosiran Treatment: A Qualitative Interview Study

Effectiveness of Patisiran Following Switch From Tafamidis for the Treatment of Hereditary Transthyretin Mediated (hATTR) Amyloidosis with Polyneuropathy

Patient Experience with Acute Hepatic Porphyria Before and After Long-Term Givosiran Treatment: A Qualitative Interview Study

Impact of Lumasiran on Long-Term Clinical Outcomes in Primary Hyperoxaluria Type 1 (PH1): a Cohort Simulation Model

Kidney Survival in Patients with Primary Hyperoxaluria Type 1 Treated with Lumasiran Compared with Historical Controls

Characteristics of Patients Treated with Givosiran in ELEVATE, a Global Observational Longitudinal Registry of Patients with Acute Hepatic Porphyria

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

HELIOS-A: Efficacy and safety of vutrisiran in patients with hATTR with polyneuropathy

Summary of the efficacy and safety results of vutrisiran compared to external placebo for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy, as published in the journal Amyloid, 2022.

Descriptive Analysis of Real-World Enrollment Data of Pediatric and Adult Patients From a Global Primary Hyperoxaluria Type 1 Registry (BONAPH1DE)

Long-term clinical outcomes of patients with acute hepatic porphyria who were not attack-free after 6 months of givosiran treatment in a subgroup analysis of the phase 3 ENVISION study

KARDIA-3: A randomized trial of zilebesiran versus placebo on top of standard care for patients with hypertension and established cardiovascular disease or high cardiovascular risk with or without chronic kidney disease

Lumasiran for Primary Hyperoxaluria Type 1: Analysis of Urinary Oxalate and eGFR Over Time in Patients With and Without Baseline Pyridoxine Use

Safety and Tolerability of Zilebesiran, an RNA Interference Therapeutic Targeting Hepatic Angiotensinogen Synthesis, in Obese Patients with Hypertension

Consistent antihypertensive efficacy of the RNA interference therapeutic zilebesiran: Subgroup results from the KARDIA-1 phase 2 study in patients with hypertension

Sustained blood pressure reduction with the RNA interference therapeutic, zilebesiran: Primary results from KARDIA 1, a phase 2 study in patients with hypertension

Multiple-Dose Results from an Ongoing Phase 1 Study of Mivelsiran, an Investigational RNA Interference Therapeutic Targeting Amyloid-Beta Precursor Protein for Alzheimer’s Disease

A Phase 1, Single Ascending Dose Study to Evaluate ALN-TTRsc04, a Next-Generation RNA Interference Therapeutic, in Healthy Participants for Potential Treatment of Transthyretin Amyloidosis

Safety and Tolerability of ALN-AGT, an RNA Interference Therapeutic Targeting Hepatic Angiotensinogen Synthesis, in Hypertensive Patients during Sodium Depletion or Irbesartan Coadministration

Durable Reductions in Circulating Angiotensinogen and Blood Pressure 6 Months after Single Doses of ALN-AGT, an RNA Interference Therapeutic Targeting Hepatic Angiotensinogen Synthesis, in Hypertensive Patients

Design and Rationale of cAPPricorn-1, a Phase 2 Study of Mivelsiran in Patients With Cerebral Amyloid Angiopathy

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