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12,372 trials

Phase 3 N=168 NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Primary: Change From Baseline in Modified Neuropathy Impairment Score Plus 7 (mNIS+7) at Week 66 — 0.2964; 25.0557 scores on a scale — p=0.00000001
Ionis Pharmaceuticals, Inc. Results Dec 2024 17.5% serious AE View details