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HELIOS-A phase 3 study of vutrisiran in hereditary transthyretin amyloidosis
NL-OMON60482Phase 3Completed
- Sponsor
- Alnylam Pharmaceuticals
- Start
- Nov 2019
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 3, open-label trial of vutrisiran in hereditary transthyretin amyloidosis (hATTR) (2 arms), sponsored by Alnylam Pharmaceuticals. Primary endpoint: Change from baseline in modified Neurologic Impairment Score +7. Started Nov 2019; status: completed.
02
Drugs
vutrisiran
03
Conditions
hereditary transthyretin amyloidosis (hATTR)
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