Search Results for "diagnosis"
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Amyloidosis Project ECHO
Virtual interactive clinic offers quarterly 1-hour sessions focused on AL (light chain) and ATTR (transthyretin)
amyloidosis, two underrecognized but increasingly manageable causes of heart failure and multisystem disease.
Virtual interactive clinic offers quarterly 1-hour sessions focused on AL (light chain) and ATTR (transthyretin) amyloidosis, two underrecognized but increasingly manageable causes of heart failure and multisystem disease.
ATTR Diagnostic and Staging Tools
This deck highlights diagnostic and staging tools for assessing patients with ATTR.
This deck highlights diagnostic and staging tools for assessing patients with ATTR.
Givosiran: Use in Patients with Prior History of Hemin Prophylaxis
Standard response letter on the use of givosiran in patients with a prior history of hemin prophylaxis from the ENVISION study.
Standard response letter on the use of givosiran in patients with a prior history of hemin prophylaxis from the ENVISION study.
ATTR-CM HELIOS-B Data Summary
ATTR-CM HELIOS-B Data Summary deck provides a comprehensive overview of the HELIOS-B results. Also includes information on ATTR-CM and the MOA of RNAi therapeutics.
ATTR-CM HELIOS-B Data Summary deck provides a comprehensive overview of the HELIOS-B results. Also includes information on ATTR-CM and the MOA of RNAi therapeutics.
Symptoms and Disease Burden of ATTR
Title: Symptoms and Disease Burden of ATTR
External description: This slide deck provides an overview of the symptoms and disease burden of ATTR.
Title: Symptoms and Disease Burden of ATTR External description: This slide deck provides an overview of the symptoms and disease burden of ATTR.
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.
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.
Givosiran: Use in Pediatric Patients
Standard response letter on givosiran and pediatric use. This medical information response contains clinical data from the ENVISION study, ELEVATE registry, and published case reports.
Standard response letter on givosiran and pediatric use. This medical information response contains clinical data from the ENVISION study, ELEVATE registry, and published case reports.
Givosiran: Use in Patients with Pre-Existing Renal Impairment
Standard response letter on the use of givosiran in patients with pre-existing renal impairment.
Standard response letter on the use of givosiran in patients with pre-existing renal impairment.
Givosiran: Post-Dose Monitoring
Standard response letter on givosiran and monitoring post-injection.
Standard response letter on givosiran and monitoring post-injection.
Lumasiran: Dialysis
Standard response letter on lumasiran and dialysis. This medical information response contains clinical data from the ILLUMINATE-C study and published case reports.
Standard response letter on lumasiran and dialysis. This medical information response contains clinical data from the ILLUMINATE-C study and published case reports.
Givosiran: Use in Patients Without an Identified Porphyria-Related Mutation
Standard response letter on the use of givosiran in patients with AHP without an identified porphyria-related genetic mutation.
Standard response letter on the use of givosiran in patients with AHP without an identified porphyria-related genetic mutation.
Givosiran: Pancreatitis
Standard response letter on givosiran and the adverse event of pancreatitis.
Standard response letter on givosiran and the adverse event of pancreatitis.
Long-term Assessment of Patients with AHP and AIP who were Not Attack-Free after 6-Months of Givosiran Treatment: A Post Hoc Subgroup Analysis of the Phase 3 ENVISION Study
An infographic depicting a post-hoc analysis of the ENVISION phase 3 study to understand the long-term clinical outcomes of patients who were not attack free after 6-months of givosiran treatment.
An infographic depicting a post-hoc analysis of the ENVISION phase 3 study to understand the long-term clinical outcomes of patients who were not attack free after 6-months of givosiran treatment.
Patisiran: Post-Orthotopic Liver Transplant
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 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.
Lumasiran: Use in Kidney Transplantation
Standard response letter on the use of lumasiran in patients who have had an isolated kidney transplant.
Standard response letter on the use of lumasiran in patients who have had an isolated kidney transplant.
Patisiran: APOLLO Study Results
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 patisiran and the APOLLO study, a phase 3 study evaluating the efficacy and safety of patisiran in patients with the hATTR-PN.
HELIOS-B Plain Language Summary
This material is a short summary of HELIOS-B primary medical and scientific publications (including peer-reviewed medical journal articles and scientific conference presentations)
This material is a short summary of HELIOS-B primary medical and scientific publications (including peer-reviewed medical journal articles and scientific conference presentations)
Lumasiran: ILLUMINATE-B Study Overview
Standard response letter on the lumasiran phase 3 clinical study ILLUMINATE-B, a study evaluating the efficacy and safety of lumasiran in infants and children less than 6 years old with PH1.
Standard response letter on the lumasiran phase 3 clinical study ILLUMINATE-B, a study evaluating the efficacy and safety of lumasiran in infants and children less than 6 years old with PH1.
Carpal Tunnel Release Surgery Handout
This handout provides an overview of two diagnostic algorithms for ATTR amyloidosis in patients with carpal tunnel syndrome.
This handout provides an overview of two diagnostic algorithms for ATTR amyloidosis in patients with carpal tunnel syndrome.
BONAPH1DE: A Prospective, Observational Study of Patients with PH1
This infographic discusses the study design, objectives, key patient criteria, and planned countries for the BONAPH1DE observational study.
This infographic discusses the study design, objectives, key patient criteria, and planned countries for the BONAPH1DE observational study.
Phase 3 HELIOS-B Study Rationale
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 slide deck highlights the Phase 3 trial design for HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy.