Search Results for "manifestation"
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Primary Results From APOLLO-B, A Phase 3 Study of Patisiran in Patients With Transthyretin-Mediated Amyloidosis With Cardiomyopathy
Alnylam Pharmaceuticals submitted a supplemental New Drug Application (sNDA) to the FDA for patisiran for the treatment of the cardiomyopathy of ATTR amyloidosis based on data from the APOLLO-B study. The FDA subsequently completed their review of the application in 2023 and issued a complete response letter (CRL).
Alnylam Pharmaceuticals submitted a supplemental New Drug Application (sNDA) to the FDA for patisiran for the treatment of the cardiomyopathy of ATTR amyloidosis based on data from the APOLLO-B study. The FDA subsequently completed their review of the application in 2023 and issued a complete response letter (CRL).
Hereditary transthyretin amyloidosis with polyneuropathy in the United States
Recommendations from a panel of experts for the diagnosis and treatment of hereditary transthyretin amyloidosis with polyneuropathy in the United States
Recommendations from a panel of experts for the diagnosis and treatment of hereditary transthyretin amyloidosis with polyneuropathy in the United States
ATTR Amyloidosis Disease State Handout
External title: ATTR Amyloidosis Disease State Handout
External summary: This handout provides an overview of ATTR amyloidosis manifestations and the diagnostic tools that can be used to assess their presentation.
External title: ATTR Amyloidosis Disease State Handout External summary: This handout provides an overview of ATTR amyloidosis manifestations and the diagnostic tools that can be used to assess their presentation.
Screening, Recognition, and Barriers to Diagnosis of ATTR
This deck highlights the screening, recognition, and barriers of diagnosis in ATTR.
This deck highlights the screening, recognition, and barriers of diagnosis in ATTR.
Hereditary ATTR Amyloidosis: A Closer Look at the V1221 Variant
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.
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.
Diagnosing AHP
AHP Diagnosis
AHP Diagnosis
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: Mechanism of Action and Chemical Properties
Standard response letter on the mechanism of action and chemical properties of givosiran.
Standard response letter on the mechanism of action and chemical properties of givosiran.
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.
ATTR Screening and Carrier Management
This deck highlights tools for ATTR screening and recommendations for the management of hATTR carriers.
This deck highlights tools for ATTR screening and recommendations for the management of hATTR carriers.
Vutrisiran: Results in Patients who had Progressed on Tafamidis in HELIOS B
Standard response letter on the use of vutrisiran in patients who had progressed on tafamidis at baseline in HELIOS-B.
Standard response letter on the use of vutrisiran in patients who had progressed on tafamidis at baseline in HELIOS-B.
The Unpredictable Progression of PH1
This handout details the potential progressive decline of kidney function in PH1, which mostly results in end-stage kidney disease.
This handout details the potential progressive decline of kidney function in PH1, which mostly results in end-stage kidney disease.
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.
Identifying AHP module
This educational module will illustrate patients with AHP, describe common AHP symptoms and summarize how patients present to the various specialties involved in managing these patients
This educational module will illustrate patients with AHP, describe common AHP symptoms and summarize how patients present to the various specialties involved in managing these patients
