Gene symbol | TTR | Synonyms | ATTR, CTS, CTS1, HEL111, HsT2651, PALB, TBPA, TTN | Type of gene | protein-coding |
Chromosome | 18 | Map location | 18q12.1 | dbXrefs | |
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 |
Therapy | siRNA |
Treatment | ALN-TTR02|ONPATTRO|patisiran |
Location approved | US, EU, UK, Japan, Canada, Switzerland, Brazil, China, Israel, Turkey, Australia |
Phase | Early_Phase1 |
Recruitment status | Recruiting |
Title | Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy |
Year | 2022 |
Country | United States|Argentina|Chile|Costa Rica |
Company sponsor | Austin Neuromuscular Center |
Other ID(s) | 001 amendment 02 |
Vector information | |||
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Cohort 1 | |||||||||
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