ARC TALKS Webinar: ATTR Amyloidosis Clinical Trials (Jan 2026) скачать в хорошем качестве

ARC TALKS Webinar: ATTR Amyloidosis Clinical Trials (Jan 2026)

Kristen Hsu, ARC’s Executive Director of Research, shared the latest updates on ATTR (transthyretin amyloidosis) clinical trials and what these developments mean for patients and families. There was also an opportunity for questions and answers. Originally recorded: January 22, 2026 0:00 — Welcome 3:34 — Introduction 5:50 — What is a Clinical Trial? 7:20 — Approaches to ATTR Amyloidosis Treatment 15:00 — ATTR Amyloidosis Clinical Trials 18:19 — ACT-EARLY (ttr stabilizer; asymptomatic carriers of ttr mutations) 25:09 — TRITON-CM (gene silencer; wild-type or hereditary attr-cm) 29:30 — TRITON-PN (gene silencer; hereditary attr-pn) 32:46 — CLEOPATTRA (anti-fibril depleter; wildtype or hereditary attr-cm) 37:27 — Upcoming Milestones in 2026 38:14 — CARDIO-TTRansform (gene silencer; wildtype or hereditary attr-cm) 40:56 — MAGNITUDE/MAGNITUDE-2 (gene editing; wildtype or hereditary attr-cm & attr-pn) 43:43 — How long does FDA approval take after Phase 3? 47:18 — Other Recruiting and Planned Clinical Studies 47:59 — How to Find Clinical Trials 51:50 — Q&A Session 51:56 — What’s the difference between TTR knockdown and stabilizer drugs? 55:02 — Are there any studies showing silencers and stabilizers are beneficial in combination or one is better than the other? 57:10 — Can you share about Attralus’ AT-02 study? 58:24 — Can you share about the Cliramitug/DepleTTR-CM (ALXN220) study? 1:00:49 — Will having an ICD (cardiac defibrilator) implanted exclude me from clinical trials? 1:01:38 — Why do trials exclude normal NT-proBNP levels? 1:03:57 — Are there trials for Wildtype ATTR without cardiac involvement? 1:06:15 — Conclusion