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Open-label extension study of long-term patisiran in familial amyloidotic polyneuropathy
2014-003877-40Phase 3Active
- Sponsor
- Alnylam Pharmaceuticals
- Start
- Mar 2015
- Modality
- Gene Therapies, 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, single-arm trial of patisiran in transthyretin amyloidosis (ATTR), familial amyloid polyneuropathy (FAP) and amyloid cardiomyopathy, sponsored by Alnylam Pharmaceuticals. Primary endpoints: Long-term safety of patisiran and Long-term efficacy of patisiran. Started Mar 2015; status: active.
02
Drugs
patisiran
03
Conditions
transthyretin amyloidosis (ATTR)familial amyloid polyneuropathy (FAP)amyloid cardiomyopathy
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