Disease · EFO_0700065
hereditary ATTR amyloidosis
6 sourcesdiseases-2026-09-18-0.1.0
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ConductScience Library. hereditary ATTR amyloidosis. Diseases, record EFO_0700065. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/EFO_0700065
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Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Other names
ATTRv amyloidosis, Amyloidosis, hereditary, transthyretin-related, TTR amyloid neuropathy, Transthyretin amyloid neuropathy, Transthyretin amyloid polyneuropathy, amyloid neuropathies, familial, familial TTR-related amyloidosis, familial amyloid neuropathy, familial amyloid polyneuropathy, familial transthyretin-related amyloidosis, hATTR, hereditary TTR amyloid polyneuropathy, hereditary TTR amyloidosis, hereditary transthyretin amyloid polyneuropathy, paramyloidosis, transthyretin amyloidosis
- Open Targets
Drugs17
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| TAFAMIDIS MEGLUMINE | CHEMBL2105675 | Small molecule | Approval | ClinicalTrials.gov, at phase 4 | 6 | 5 | 5 | Drugs and targets |
| ACORAMIDIS | CHEMBL3940890 | Small molecule | Approval | EMA Human Drugs | 3 | 2 | 2 | Drugs and targets |
| EPLONTERSEN | CHEMBL4594334 | Oligonucleotide | Approval | EMA Human Drugs | 3 | 2 | 2 | Drugs and targets |
| VUTRISIRAN | CHEMBL4594511 | Oligonucleotide | Approval | EMA Human Drugs and FDA | 2 | 0 | 0 | Drugs and targets |
| DIFLUNISAL | CHEMBL898 | Small molecule | Approval | EMA Human Drugs | 1 | 0 | 0 | Drugs and targets |
| EPLONTERSEN SODIUM | CHEMBL4594335 | Oligonucleotide | Approval | FDA | 1 | 0 | 0 | Drugs and targets |
| INOTERSEN SODIUM | CHEMBL4297537 | Oligonucleotide | Approval | FDA | 1 | 0 | 0 | Drugs and targets |
| PATISIRAN SODIUM | CHEMBL4297536 | Oligonucleotide | Approval | FDA | 1 | 0 | 0 | Drugs and targets |
| VUTRISIRAN SODIUM | CHEMBL5095303 | Oligonucleotide | Approval | FDA | 1 | 0 | 0 | Drugs and targets |
| TAFAMIDIS | CHEMBL2103837 | Small molecule | Phase 3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
1 to 10 of 17
See all 17 drugsTargets9
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| TTR | Transthyretin | 11 | Transthyretin mRNA RNAi inhibitor; Transthyretin mRNA antisense inhibitor; Transthyretin mRNA rnai inhibitor; Transthyretin stabiliser | Approval | https://conductscience.org/library/genes/7276 | Drugs and targets |
| COMT | Catechol O-methyltransferase | 1 | Catechol O-methyltransferase inhibitor | Early phase 1 | https://conductscience.org/library/genes/1312 | Drugs and targets |
| MMP1 | Interstitial collagenase | 1 | Matrix metalloproteinase-1 inhibitor | Phase 2 | https://conductscience.org/library/genes/4312 | Drugs and targets |
| MMP13 | Collagenase 3 | 1 | Matrix metalloproteinase 13 inhibitor | Phase 2 | https://conductscience.org/library/genes/4322 | Drugs and targets |
| MMP7 | Matrilysin | 1 | Matrix metalloproteinase 7 inhibitor | Phase 2 | https://conductscience.org/library/genes/4316 | Drugs and targets |
| MMP8 | Neutrophil collagenase | 1 | Matrix metalloproteinase 8 inhibitor | Phase 2 | https://conductscience.org/library/genes/4317 | Drugs and targets |
| NR1H4 | Bile acid receptor | 1 | Bile acid receptor FXR agonist | Phase 2 | https://conductscience.org/library/genes/9971 | Drugs and targets |
| PTGS1 | Cyclooxygenase | 1 | Cyclooxygenase inhibitor | Approval | https://conductscience.org/library/genes/5742 | Drugs and targets |
| PTGS2 | Cyclooxygenase | 1 | Cyclooxygenase inhibitor | Approval | https://conductscience.org/library/genes/5743 | Drugs and targets |
Trials20
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT01960348 | APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis | Phase 3 | 225 | 2018-09-06 | TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related | PATISIRAN | Its record names the disease | ClinicalTrials.gov |
| NCT01737398 | Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy | Phase 2/3 | 173 | 2019-01-23 | FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis | INOTERSEN | Its record names the disease | ClinicalTrials.gov |
| NCT02175004 | Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP) | Phase 3 | 135 | 2023-02-09 | FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis | INOTERSEN | Its record names the disease | ClinicalTrials.gov |
| NCT00409175 | Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis | Phase 2/3 | 128 | 2012-12-17 | Familial Amyloid Polyneuropathy | TAFAMIDIS MEGLUMINE | Its record names the disease | ClinicalTrials.gov |
| NCT00925002 | Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis | Phase 3 | 93 | 2021-07-29 | ATTR-PN | TAFAMIDIS | Its record names the disease | ClinicalTrials.gov |
| NCT00791492 | An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy | Phase 2/3 | 86 | 2012-12-17 | Familial Amyloid Polyneuropathy, ATTR-PN | TAFAMIDIS | Its record names the disease | ClinicalTrials.gov |
| NCT01617967 | Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis | Phase 2 | 29 | 2018-11-21 | TTR-mediated Amyloidosis | PATISIRAN | Its record names the disease | ClinicalTrials.gov |
| NCT00630864 | The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis | Phase 2 | 21 | 2012-12-17 | Transthyretin-associated Amyloidosis With Polyneuropathy | TAFAMIDIS MEGLUMINE | Its record names the disease | ClinicalTrials.gov |
| NCT04828993 | The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients | Phase 4 | 15 | 2024-03-12 | Transthyretin Amyloid Polyneuropathy (ATTR-PN) | TAFAMIDIS MEGLUMINE | Its record names the disease | ClinicalTrials.gov |
| NCT01435655 | The Effect Of Tafamidis For The Transthyretin Amyloid Polyneuropathy Patients With V30M Or Non-V30M Transthyretin | Phase 3 | 10 | 2015-09-09 | Transthyretin Familial Amyloid Polyneuropathy | TAFAMIDIS | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 20
See all 20 trialsComparisons in its trials11
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Patisiran (ALN-TTR02) vs Placebo: deaths from any cause | 7 of 148 | 6 of 77 | 225 | NCT01960348 | Trial comparisons |
| Patisiran (ALN-TTR02) vs Placebo: serious adverse events | 54 of 148 | 31 of 77 | 225 | NCT01960348 | Trial comparisons |
| Inotersen vs Placebo: deaths from any cause | 5 of 112 | 0 of 60 | 172 | NCT01737398 | Trial comparisons |
| Inotersen vs Placebo: serious adverse events | 36 of 112 | 13 of 60 | 172 | NCT01737398 | Trial comparisons |
| Previous Inotersen-Inotersen 300 mg vs Previous Placebo-Inotersen 300 mg: deaths from any cause | 15 of 85 | 2 of 50 | 135 | NCT02175004 | Trial comparisons |
| Previous Inotersen-Inotersen 300 mg vs Previous Placebo-Inotersen 300 mg: serious adverse events | 46 of 85 | 18 of 50 | 135 | NCT02175004 | Trial comparisons |
| Tafamidis vs Placebo: serious adverse events | 6 of 65 | 5 of 63 | 128 | NCT00409175 | Trial comparisons |
| Tafamidis-Tafamidis vs Placebo-Tafamidis: Number of Participants With Treatment-emergent Adverse Events Greater Than or Equal to Grade 3 | 6 of 44 | 4 of 41 | 85 | NCT00791492 | Trial comparisons |
| Tafamidis-Tafamidis vs Placebo-Tafamidis: Number of Participants With Treatment-emergent Serious Adverse Events (SAEs) | 5 of 44 | 4 of 41 | 85 | NCT00791492 | Trial comparisons |
| Tafamidis-Tafamidis vs Placebo-Tafamidis: Number of Participants Who Discontinued Due to Clinical or Laboratory Adverse Events | 0 of 44 | 0 of 41 | 85 | NCT00791492 | Trial comparisons |
1 to 10 of 11
See all 11 comparisons in its trialsPapers from its trials35
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
| PMID | Listed as | Trials listing it | Source |
|---|---|---|---|
| 29972757 | RESULT | 1 | PubMed |
| 41937511 | DERIVED | 1 | PubMed |
| 41913562 | DERIVED | 1 | PubMed |
| 40108078 | DERIVED | 1 | PubMed |
| 38548524 | DERIVED | 1 | PubMed |
| 38521883 | DERIVED | 1 | PubMed |
| 38065307 | DERIVED | 1 | PubMed |
| 36120830 | DERIVED | 1 | PubMed |
| 35908242 | DERIVED | 1 | PubMed |
| 35799473 | DERIVED | 2 | PubMed |
1 to 10 of 35
See all 35 papers from its trialsData dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/EFO_0700065
GET https://conductscience.org/api/diseases/v1/EFO_0700065/papers?page=2