Eplontersen for Transthyretin Amyloid Cardiomyopathy.
BACKGROUND: Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, life-threatening disease caused by deposition of misfolded transthyretin (TTR). RESULTS: A total of 1432 patients underwent randomization and received at least one dose of the assigned intervention (715 patients in the eplontersen group and 717 in the placebo group).
BACKGROUND: Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, life-threatening disease caused by deposition of misfolded transthyretin (TTR). RESULTS: A total of 1432 patients underwent randomization and received at least one dose of the assigned intervention (715 patients in the eplontersen group and 717 in the placebo group).
What the study was
- Study design
- Phase 3 clinical trial
- Sample size
- 1432
- Category
- Drug Development
- Maturity
- Potentially Practice-Changing
- Journal
- The New England journal of medicine
Why it surfaced
High-priority Rare diseases with high unmet need study: novel treatment potential warrants immediate pipeline escalation.
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