| 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 | GTC1853 |
| Trial ID | NCT03400098 |
| Disease | Transthyretin Amyloidosis |
| Altered gene | TTR |
| Therapeutic/Target gene | Target gene |
| Therapy | ASO |
| Treatment | ISIS 416858|IONIS-FXIRX|FXI-ASO|ISIS-FXI Rx|BAY2306001||BAY2306001|Fesomersen |
| Recruitment status | Approved For Marketing |
| Title | Expanded Access Program for Inotersen (ISIS 420915) in Patients With Hereditary Transthyretin Amyloidosis (hATTR) |
| Year | 2018 |
| Country | United States |
| Company sponsor | Ionis Pharmaceuticals, Inc. |
| Other ID(s) | ISIS 420915-CS5 |
| Vector information | |||
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| Cohort 1 | |||||
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