FDA Issues Complete Response Letter for Patisiran for Treatment of Cardiomyopathy of ATTR Amyloidosis
BREAKING NEWS: The U.S. Food and Drug Administration has issued a complete response letter for Alnylam Pharmaceuticals' Patisiran, an investigational RNAi therapeutic for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis. This letter denies approval for the treatment of...
Taming Wild-Type Amyloidosis — FAQ
Leading cardiologist Dr. Martha Grogan was our guest speaker for Taming Wild-Type, our recent ARC Talks webinar focused on wild-type transthyretin amyloidosis (ATTRwt). In the presentation, Dr. Grogan, the Founder and Director of the Cardiac Amyloid Clinic at the Mayo...
Vutrisiran receives Fast Track Designation from FDA
Alnylam has submitted a new drug application (NDA) to the FDA for vutrisiran, which could bring another treatment option to patients with ATTR amyloidosis. Vutrisiran received “Fast Track” Designation, which is a process designed to expedite the review of drugs...
Early Findings from Novel CRISPR Therapy Show Promise for hATTR Amyloidosis
July 7, 2021 CRISPR technology offers a cutting-edge approach to gene-editing. New clinical findings from Intellia’s Phase 1 trial have demonstrated for the first time that CRISPR can be administered by infusion to target specific cells within the body. (more…)
Report from ISA Meeting 2020
A Report from ISA 2020 Every 2 years, amyloidosis researchers and physicians from all around the world engage in a week-long meeting, the International Symposium on Amyloidosis (ISA). (more…)