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Long-term extension study of patisiran in familial amyloidotic polyneuropathy
jRCT2080224174Completed
- Sponsor
- Alnylam Pharmaceuticals
- Start
- Dec 2018
- 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
01
Overview
Open-label, single-arm trial of patisiran in familial amyloid polyneuropathy (FAP) and hereditary transthyretin amyloidosis (hATTR), sponsored by Alnylam Pharmaceuticals. Started Dec 2018; status: completed.
02
Drugs
patisiran
03
Conditions
familial amyloid polyneuropathy (FAP)hereditary transthyretin amyloidosis (hATTR)
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