Basic Information

Gene symbol TTR Synonyms ATTR, CTS, CTS1, HEL111, HsT2651, PALB, TBPA, TTN Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas
Description transthyretin

GTO ID GTC2776
Trial ID NCT05023889
Disease Polyneuropathy | Wild-Type Amyloidosis
Altered gene TTR
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-TTR02|ONPATTRO|patisiran
Location approved US, EU, UK, Japan, Canada, Switzerland, Brazil, China, Israel, Turkey, Australia
PhaseEarly_Phase1
Recruitment statusRecruiting
TitleSpectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
Year2022
CountryUnited States|Argentina|Chile|Costa Rica
Company sponsorAustin Neuromuscular Center
Other ID(s)001 amendment 02
Vector information
Vectorlipid nanoparticle

Clinical Result

Cohort 1
Administration route intravenous infusion
Dosage patisiran, 0.3 mg/kg once every 21 days, over 70 minutes
Pts 10
Age Adult, Older_Adult

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