Search Results for "amyloidosis"

Standard response letter on vutrisiran and the changes in serum TTR levels during the HELIOS-A and HELIOS-B studies.

Standard response letter on switching from TTR stabilizers (tafamidis, acoramidis, or diflunisal) to vutrisiran.

Standard response letter on patisiran and changes in serum TTR levels observed in the Phase 2 OLE, APOLLO, APOLLO-B, and HELIOS-A studies.

Understanding ATTR amlyloidosis: This slide deck highlights an overview of ATTR amyloidosis pathophysiology, symptom onset and prevalence

Standard response letter on vutrisiran and results from an 18-month analysis of the randomized treatment extension period of HELIOS-A.

Standard Response Letter on vutrisiran and its pharmacokinetic and pharmacodynamic properties.

Standard response letter on transitioning between patisiran and vutrisiran therapy.

This slide deck presents of overview of ATTR amyloidosis, covering pathophysiology, disease manifestations and the clinical burden of ATTR-CM.

Standard response letter on patisiran and vitamin A levels.

This slide deck provides disease education on ATTR amyloidosis, focusing on the musculoskeletal manifestations associated with the disease and their importance for early diagnosis and patient care.

Standard response letter on the mechanism of action of vutrisiran.

This material is a short summary of HELIOS-B primary medical and scientific publications (including peer-reviewed medical journal articles and scientific conference presentations)

Standard response letter on filters used for the preparation and infusion of patisiran.

This slide deck highlights the Phase 3 trial design for HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy.

Standard response letter on the phase 1 study of nucresiran.

This interactive PDF provides an overview of ATTR-CM and RNAi therapeutics, and offers a deep dive into the vutrisiran data in ATTR-CM from the HELIOS-B trial, including the primary and additional analyses. Alnylam's ongoing commitment to ATTR amyloidosis is reflected in a summary of the current pipeline and TRITON-CM study design.

Standard response letter on vutrisiran and results from the HELIOS-A study evaluating efficacy and safety of vutrisiran in patients with the polyneuropathy of hATTR.

Standard response letter on patisiran and the open-label phase 3b study evaluating the efficacy, safety and PK of patisiran in patients with hATTR who had polyneuropathy progression post-OLT. Additional summaries of case reports are also included.

Standard response letter on patisiran and use in patients with renal impairment. The letter contains information from a post-hoc analysis in patients with ATTR and CKD across the APOLLO, APOLLO-B, Phase 3b Post-OLT, Phase 2 OLE, and Global OLE studies, as well as relevant information from a global safety database.

Standard response letter on patisiran and the APOLLO study, a phase 3 study evaluating the efficacy and safety of patisiran in patients with the hATTR-PN.

Standard response letter on the efficacy and safety of vutrisiran and patisiran, summarizing data from the HELIOS-A study and the APOLLO study.