Search Results for "amyloidosis"

Standard response letter on the mechanism of action of patisiran.

This deck highlights the red flag symptoms of ATTR and the tools to help achieve an accurate diagnosis.

V122I is the most common variant of hATTR amyloidosis in the United States and is most prevalent in patients of African American descent. Compared to other variants, patients with the V122I variant experience significant disease burden and poorer outcomes.

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.

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

This deck highlights data from the Phase 3 HELIOS-A trial of vutrisiran in patients with hATTR-PN.

Standard response letter on patisiran and pregnancy & lactation. This letter contains information on preclinical toxicology studies and the APOLLO, APOLLO-B, and HELIOS-A studies.

Standard response letter on patisiran and the APOLLO-B study, a study evaluating the efficacy and safety of patisiran in patients with ATTR with cardiomyopathy, including both hATTR and wtATTR.

This deck highlights early indicators of ATTR and systemic manifestations.

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

This deck highlights tools for ATTR screening and recommendations for the management of hATTR carriers.

Standard response letter on vutrisiran and vitamin A levels, including information on supplementation.

Standard response letter on the cross-reactivity between vutrisiran and patisiran.

Standard response letter on the preparation and administration of patisiran.

Standard response letter on patisiran and product stability. The letter includes information on the chemical, physical and photostability of the active substance, patisiran, and the diluted patisiran solution.

Standard response letter on patisiran and total dilution volume. This letter contains information on a stability study and the APOLLO, APOLLO-B, and Global OLE studies.

Standard response letter on patisiran and temperature excursions. The letter includes information on the impact of temperature excursions not lower than -4°C and up to 40°C on the stability of patisiran.

Standard response letter on use of mRNA COVID-19 vaccines in patients receiving vutrisiran.

Standard response letter on patisiran and use in patients with heart transplant.

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

External title: Patisiran RWE in hATTR Amyloidosis External description: This slide deck highlights real-world evidence (RWE) data on the use of patisiran in patients with hATTR amyloidosis, including patients with polyneuropathy, cardiomyopathy, solid organ transplant, concomitant treatment or treatment switch.

Standard response letter on transitioning from an antisense oligonucleotide to vutrisiran.

Standard response letter on patisiran and compatibility with commonly used infusion components based on a compatibility and stability study.

Standard response letter on the APOLLO-B OLE study of patisiran.

Standard response letter on hospitalization and mortality data from patisiran clinical trials.