| 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 | GTC2274 |
| Trial ID | NCT04201418 |
| Disease | Polyneuropathy | Transthyretin 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 |
| Recruitment status | Completed |
| Title | A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation |
| Year | 2019 |
| Country | United States |
| Company sponsor | Alnylam Pharmaceuticals |
| Other ID(s) | ALN-TTR02-012 |
| Vector information | |||
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| Cohort1: naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy | |||||
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| Cohort2: currently on commercial patisiran therapy for less than 12 months | |||||
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| Cohort3: commercial patisiran therapy for at least 12 months prior to study enrollment | |||||
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