Groundbreaking News for ATTR Cardiac Amyloidosis Community
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Every year around Rare Disease Day (February 28th), The EveryLife Foundation hosts Rare Disease Week on Capitol Hill. The event aims to bring together the rare disease community to educate them federal legislative issues and give them the chance to...
Pfizer’s 2018 Global ASPIRE TTR Amyloidosis Competitive Grants Program
Pfizer is pleased to announce the 2018 Global ASPIRE Transthyretin (TTR) Amyloidosis Competitive Research Grant Awards for Junior Investigators. The Global ASPIRE TTR Amyloidosis program underscores Pfizer’s commitment to supporting investigators with an interest in advancing their academic research in...
Can’t Attend Rare Disease Week? Five Ways to Participate Remotely
Rare disease day is on February 28th and every year the Rare Disease Legislative Advocates, a program of the EveryLife Foundation for Rare Diseases, hosts Rare Disease Week on Capitol Hill. The entire week provides a unique opportunity to raise awareness...
Amyloidosis experts from academia, industry and the FDA met last week for the Amyloidosis Research Consortium Inaugural Research Strategy Roundtable in Miami. The meeting, which brought together representatives from across the research continuum for the first time, was designed to...
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In this ARC Talks Webinar, Dr. Morie Gertz gave a 101 overview of clinical trials, including consideration in how they are designed, and the phases needed for drug development. Dr. Gertz also discussed current clinical trials that are evaluating the next generation of drugs for the treatment of AL and ATTR amyloidosis.
Original Presentation Date:
May 25, 2023
Key Webinar Highlights:
0:00 — Introduction
3:10 — Start of Dr. Gertz’s presentation
4:30 — The Necessity of Clinical Trials
13:50 — The Phases of Clinical Trials
23:23 — Definition of An Open Label Extension
24:47 — The Study of Vyndaquel for ATTR-CM Patients
26:18 — The Study of Drug Therapies for ATTR-PN/hATTR patients