The Inflation Reduction Act: The Devil is in the Details for Patients with Rare Diseases
David Gibson, PhD, CMPP Director of Strategic Programs, Engagement, and Communications Amyloidosis Research Consortium The Inflation Reduction Act (IRA), signed into law on August 16, 2022, tackled a wide range of issues from tax reform, energy security, environmental and...
FDA Approves WAINUA™ (Eplontersen) for Treatment of Hereditary ATTR Amyloidosis Polyneuropathy
BREAKING NEWS: The U.S. Food and Drug Administration has approved AstraZeneca and Ionis’ WAINUA™ (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. WAINUA™ (eplontersen) is a Ligand-Conjugated Antisense...
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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...
NIH Funds First Step Towards Advancing ATTR Amyloidosis Drug Development
The NIH has awarded the Amyloidosis Research Consortium $40,000 to support the organization's Amyloidosis Forum meeting on "Advancing Drug Development in ATTR Amyloidosis in an Evolving Treatment Landscape." Formed in 2019, The Amyloidosis Forum (https://amyloidosisforum.org) has been developed through a...
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This webinar “Coffee Chat” style webinar is your chance to learn about the different programs and projects ARC has been working on in 2025! Hear directly from ARC staff how we are supporting patients and helping shape the future of amyloidosis. There was also an opportunity for questions and answers.