Search Results for "therapeutic"
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Hereditary ATTR Amyloidosis: A Closer Look at the V122I Variant External
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.
RNAi Therapeutics: How Do They Work?
This video highlights the MOA of RNAi and how small interfering RNA (siRNA) are used to harness the RNA-induced silencing complex (RISC) to regulate gene expression. This resource is intended to be viewed in its entirety to support scientific exchange and is not intended as recommendations for clinical practice. For further information, please see RNAiScience.com to connect with a Medical Science Liaison, submit a medical information request, or access other Alnylam medical education resources.
This video highlights the MOA of RNAi and how small interfering RNA (siRNA) are used to harness the RNA-induced silencing complex (RISC) to regulate gene expression. This resource is intended to be viewed in its entirety to support scientific exchange and is not intended as recommendations for clinical practice. For further information, please see RNAiScience.com to connect with a Medical Science Liaison, submit a medical information request, or access other Alnylam medical education resources.
RNAi Data Navigator: Focus on Vutrisiran
Use this interactive tool to learn more about ATTR, RNAi therapeutics, findings from the HELIOS B study in ATTR CM, findings from the HELIOS A study in hATTR PN, and Alnylam’s commitment to ATTR. HELIOS-B data include outcomes on survival, functional capacity, quality of life, CV events, exploratory measures of cardiac biomarkers, structure and function, post hoc extracardiac analyses, and safety. HELIOS-A data include outcomes on neuropathy impairment, quality of life, gait speed, nutritional status, disability, and safety. Navigate using the forward and backward controls. For optimal viewing, enter full-screen mode.
Use this interactive tool to learn more about ATTR, RNAi therapeutics, findings from the HELIOS B study in ATTR CM, findings from the HELIOS A study in hATTR PN, and Alnylam’s commitment to ATTR. HELIOS-B data include outcomes on survival, functional capacity, quality of life, CV events, exploratory measures of cardiac biomarkers, structure and function, post hoc extracardiac analyses, and safety. HELIOS-A data include outcomes on neuropathy impairment, quality of life, gait speed, nutritional status, disability, and safety. Navigate using the forward and backward controls. For optimal viewing, enter full-screen mode.
Summary of RNA Interference
An overview of RNAi discovery, how RNAi works, and features of RNAi therapeutics
An overview of RNAi discovery, how RNAi works, and features of RNAi therapeutics
The Science of RNA interference (RNAi)
This slide deck provides an overview of the science of RNA interference (RNAi), including mechanism of action and delivery platforms.
This slide deck provides an overview of the science of RNA interference (RNAi), including mechanism of action and delivery platforms.
Zilebesiran: Mechanism of Action
Standard response letter on the mechanism of action of zilebesiran.
Standard response letter on the mechanism of action of zilebesiran.
Vutrisiran: Use with mRNA COVID-19 Vaccines
Standard response letter on use of mRNA COVID-19 vaccines in patients receiving vutrisiran.
Standard response letter on use of mRNA COVID-19 vaccines in patients receiving vutrisiran.
ALN-HTT02: Phase 1b Study
Standard response letter on the phase 1 study of ALN-HTT02.
Standard response letter on the phase 1 study of ALN-HTT02.
Vutrisiran: Technetium Scintigraphy
Standard response letter on vutrisiran and technetium scintigraphy data assessed in a subset of patients from the HELIOS-A study.
Standard response letter on vutrisiran and technetium scintigraphy data assessed in a subset of patients from the HELIOS-A study.