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ARC’s own Kristen Hsu presented the most current updates for the clinical trial landscape, with a focus on ATTR amyloidosis. Kristen will also explored the direction future research.

The webinar concluded with a Q and A session.

Original Presentation Date:
June 20, 2024

Download Kristen’s Slides →

0:00 — Intro

4:19 — What is a Clinical Trial?

8:06 — Should I Participate in a Trial?

10:13 — ATTR Amyloidosis Clinical Trials

14:00 — Clinical Trials Currently Recruiting

15:29 — MAGNITUDE (NTLA-2001; CRISPR Gene Editing)

21:36 — DepleTTR-CM (ALXN2220; Amyloid Depleter)

24:35 — Ongoing Clinical Trials

24:57 — ATTRibute-CM (Acoramidis; TTR Stabilizer)

26:16 — HELIOS-B (Vutrisiran; Gene Silencer)

27:25 — CARDIO-TTRansform (Eplontersen; Gene Silencer)

28:38 — Coramitug (NNC6019-0001; Anti-Amyloid Fibril)

29:39 — AT-02 (Pan-Amyloid Anti-Fibril)

31:17 — Planned Future Trials

33:14 — Other Clinical Trials

37:25 — How to Find Clinical Trials

42:00 — Q&A Session

42:33 — What is the upcoming study for hereditary ATTR carriers?

43:12 — What is a “knockdown” TTR drug?

45:09 — Does race have an impact on treatment?

46:24 — Are there studies looking at early diagnosis/natural progression of ATTR?

48:35 — How can people outside of the US participate in studies and trials?

49:44 — Are there clinical trials looking to prevent heart involvement?

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