Search Results for "RNAi"

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Efficacy and Safety of Vutrisiran in ATTR-CM Across the Spectrum of Age

Clinical outcomes in patients with acute hepatic porphyria treated with givosiran who stopped hemin prophylaxis at study entry: Post hoc analyses from the phase 3 ENVISION study through month 36

Vutrisiran In Patients With Transthyretin Amyloidosis with Cardiomyopathy In HELIOS-B Who Had Progressed On Tafamidis

Impact of Patisiran on Health Status and Quality of Life in Patients with Transthyretin Cardiac Amyloidosis

Real-World Treatment Patterns in Participants with Transthyretin Amyloidosis with Polyneuropathy Enrolled in the ConTTRibute Registry

Clinical Outcomes in Patients with Acute Hepatic Porphyria Treated with Givosiran Who Stopped Hemin Prophylaxis at Study Entry: Post hoc Analyses from the Phase 3 ENVISION Study Through Month 36

Long-term Effects of Patisiran on Survival and Cardiac Parameters in Patients with Transthyretin-mediated Cardiac Amyloidosis: Post Hoc Analysis of APOLLO-B and Cardiac Subpopulation of APOLLO OLE

Lumasiran for Primary Hyperoxaluria Type 1: Analysis of Urinary Oxalate and Estimated Glomerular Filtration Rate Over Time in Patients by Genotype

Kidney Function and Isolated Kidney Transplant Outcomes in Primary Hyperoxaluria Type 1 Treated with Long-term Lumasiran

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

Primary Results from APOLLO-B Open-label Extension Study of Patisiran in Patients with Transthyretin Cardiac Amyloidosis

Evaluation of Disease Progression in Patients with ATTR Amyloidosis with Cardiomyopathy Following Treatment with Patisiran: Post hoc Analysis of the APOLLO-B Study

Mivelsiran: Phase 1 Study

Standard response letter on the phase 1 study of mivelsiran.

Insights from the phase 3 HELIOS-A study of Vutrisiran in patients with hATTR-PN.

This deck highlights data from the phase 3 HELIOS-A trial, including exploratory cardiac endpoints and post-hoc analyses.

Identifying and Characterizing Comorbid Cerebral Amyloid Angiopathy in ADNI Participants using Boston Criteria Version 2.0

Givosiran: Phase 1/2 Open-Label Extension

Standard response letter on givosiran and the Phase 1/2 open-label extension study, an extension of the Phase 1 clinical study to evaluate the long term safety and tolerability of givosiran in patients with AIP for up to 48 months.

Understanding AHP Infographic

This infographic about AHP describes the types of the disease, including AIP, pathophysiology, clinical presentation of acute and chronic symptoms, epidemiology, and diagnosis

Patisiran: Immunogenicity

Standard response letter on patisiran and the percentage of patients in patisiran clinical studies who tested positive for anti-drug antibodies.

Givosiran: Injection Site Reactions

Standard response letter on givosiran and injection site reactions during the phase 1, phase 1/2 open-label extension, and phase 3 ENVISION studies.

Cardiac Function, Clinical Outcomes and Effect of Vutrisiran in Transthyretin Amyloid Cardiomyopathy - the HELIOS-B Trial

Impact of givosiran treatment on symptoms between acute attacks of acute hepatic porphyria as assessed by dimension-level analysis of EQ-5D data from the ENVISION study

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

NfL as a Biomarker in hATTR Amyloidosis Infographic.

Infographic companion to the article by Sato et al, "Neurofilament light chain as a biomarker for monitoring response to change in treatment in hereditary ATTR amyloidosis", published in the journal Amyloid, 2023.

Long-Term Efficacy and Safety in the Phase 3 ILLUMINATE-B Trial of Lumasiran for Primary Hyperoxaluria Type 1 in Infants and Young Children

Kidney Function and Isolated Kidney Transplant Outcomes in Primary Hyperoxaluria Type 1 Treated Long-term With Lumasiran

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Search Results for "RNAi"

Mivelsiran: Phase 1 Study Standard response letter on the phase 1 study of mivelsiran.

Insights from the phase 3 HELIOS-A study of Vutrisiran in patients with hATTR-PN. This deck highlights data from the phase 3 HELIOS-A trial, including exploratory cardiac endpoints and post-hoc analyses.

Givosiran: Phase 1/2 Open-Label Extension Standard response letter on givosiran and the Phase 1/2 open-label extension study, an extension of the Phase 1 clinical study to evaluate the long term safety and tolerability of givosiran in patients with AIP for up to 48 months.

Understanding AHP Infographic This infographic about AHP describes the types of the disease, including AIP, pathophysiology, clinical presentation of acute and chronic symptoms, epidemiology, and diagnosis

Patisiran: Immunogenicity Standard response letter on patisiran and the percentage of patients in patisiran clinical studies who tested positive for anti-drug antibodies.

Givosiran: Injection Site Reactions Standard response letter on givosiran and injection site reactions during the phase 1, phase 1/2 open-label extension, and phase 3 ENVISION studies.

NfL as a Biomarker in hATTR Amyloidosis Infographic. Infographic companion to the article by Sato et al, "Neurofilament light chain as a biomarker for monitoring response to change in treatment in hereditary ATTR amyloidosis", published in the journal Amyloid, 2023.