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Rare and hereditary disorders
Hereditary ATTR amyloidosis
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Publications and studies (1285)
- Endothelial glycocalyx disruption and early renal tubular injury in hereditary transthyretin Amyloidosis with dysautonomia. (2026/06/01) ♡
- Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients. (2026/06/01) ♡
- Visceral technetium-99 m-labeled pyrophosphate uptake in patients with wild-type transthyretin cardiac amyloidosis. (2026/06/01) ♡
- Timeliness of Transthyretin Cardiac Amyloidosis Diagnosis in the Medicare Population. (2026/06/01) ♡
- Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Rationale and Design of CARDIO-TTRansform, a Phase 3 Trial of Eplontersen in Transthyretin Amyloid Cardiomyopathy. (2026/06/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Clinical Diabetic Peripheral Neuropathy: Can It Be Reversed? Arguments for and Against From a NEUROdiab Debate. (2026/06/01) ♡
- Multimodal Artificial Intelligence for Cardiac Amyloidosis Diagnosis: Integrating Echocardiography With Clinical and Laboratory Data for Improved Detection. (2026/06/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Deep Phenotyping of F64L Mutation in a Multicentric Cohort of Patisiran-Treated Hereditary Transthyretin Amyloidosis Patients (Patisiranitaly). (2026/06/01) ♡
- Context-of-use-Guided Development and Validation of a Transthyretin Immunoassay: A Framework for Biomarker Assay Design. (2026/06/01) ♡
- A Quantitative Assessment of Upper Limb Motor Function Across Disease Stages in Hereditary Transthyretin Amyloidosis. (2026/06/01) ♡
- Evaluation of the Current ATTR-CM Treatment Landscape via a Mathematical Model of TTR Dissociation and Amyloid Formation. (2026/06/01) ♡
- Systematic reviewiAll research on one question is searched according to fixed rules and compared side by side, so that no studies with poor outcomes are missed. Pharmacological Interventions for Hereditary Transthyretin-Related Amyloidosis With Polyneuropathy: Systematic Review and Network Meta-Analysis. (2026/06/01) ♡
- A pilot qualitative study of patient understanding and perceptions of genetic counselors and cascade testing in the context of transthyretin cardiac amyloidosis. (2026/06/01) ♡
- Significance of aberrant nerve conduction in hereditary transthyretin amyloidosis. (2026/06/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Redefining optimal medical therapy for heart failure in amyloid transthyretin cardiomyopathy. (2026/06/01) ♡
- The role of nutritional status, measured by serum albumin, as a prognostic factor in wild-type transthyretin amyloidosis. (2026/06/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Relevant Adverse Events and Drug Discontinuation of Sacubitril/Valsartan in Patients with Transthyretin Amyloid Cardiomyopathy - Insights from the REVIEW-HF Registry. (2026/05/30) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Biomolecular mechanisms of cardiac amyloidosis and its cardiovascular pathological basis. (2026/05/29) ♡
- Severe myocardial involvement in oculoleptomeningeal hereditary transthyretin amyloidosis. (2026/05/22) ♡
- Long-read Oxford Nanopore sequencing enables rapid, cost-effective, and comprehensive TTR genetic testing for hereditary transthyretin amyloidosis. (2026/05/20) ♡
- Three-Dimensional Visualization and Proteomic Analysis of Human Cardiac Transthyretin Amyloidosis Tissue Reveals Microangiopathy and Capillary Occlusion. (2026/05/19) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Pathogenic Characterization of a Novel G47R Transthyretin Mutation in Early-Onset Amyloid Cardiomyopathy. (2026/05/19) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Wearables for Telemonitoring in ATTR-Amyloidosis: Current Perspectives. (2026/05/11) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Clinical Practice and Diagnostic Trends in Hereditary Transthyretin Amyloidosis: A 25-Year Observational Study. (2026/05/07) ♡
- Right Atrial Function as a Novel Predictor for New-Onset Atrial Fibrillation in Transthyretin Amyloid Cardiomyopathy. (2026/05/05) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Bone Tracers for Transthyretin Amyloid Cardiomyopathy: Are [(99m)Tc]Tc-DPD and [(99m)Tc]Tc-HMDP Equivalent? (2026/05/05) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. [A clinical case of a mixed variant (cardiomyopathy and polyneuropathy) of hereditary transthyretin amyloidosis]. (2026/05/02) ♡
- Unique TTR Variants D38A and M13dup Among Korean Patients with Hereditary Transthyretin Amyloidosis: A Retrospective Single-Center Cohort Study. (2026/05/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. CMR-based assessment of long-term effects of tafamidis in patients with cardiac transthyretin amyloidosis. (2026/05/01) ♡
- Left ventriculo-arterial coupling in a contemporary cohort of patients with wild-type transthyretin cardiac amyloidosis treated with tafamidis. (2026/05/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Cardiac transthyretin amyloidosis in aortic valve replacement: RAISE score performance in the postoperative setting. (2026/05/01) ♡
- Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Long-Term Durability of Acoramidis Efficacy in Transthyretin Amyloid Cardiomyopathy: Open-Label Extension of the ATTRibute-CM Randomized Clinical Trial. (2026/05/01) ♡
- Kinetics of Technetium-Labeled Cardiac Amyloid Radionuclide Imaging. (2026/05/01) ♡
- Neuropathic involvement in wild-type transthyretin amyloidosis. (2026/05/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Multidimensional Assessment of Disease Progression in a Contemporary Population With Transthyretin Amyloid Cardiomyopathy: Integrating Clinical Parameters, Biomarkers, Functionality, and Patient-Reported Outcomes. (2026/05/01) ♡
- Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy and Renal Impairment: Analyses From HELIOS-B. (2026/05/01) ♡
- Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Acoramidis, Serum Transthyretin, and Cardiovascular Outcomes in Transthyretin Amyloid Cardiomyopathy: Insights From the ATTRibute-CM Trial. (2026/05/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Quantitative cardiac amyloid radionuclide imaging. (2026/05/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Cardiac amyloid radionuclide imaging: Global perspective and future priorities. (2026/05/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. An era of precision: Emerging, research, and adjunct radiotracers for nuclear imaging of cardiac amyloidosis. (2026/05/01) ♡
- Associated diseases and diuretic dosage to predict mortality in transthyretin amyloid cardiomyopathy. (2026/05/01) ♡
- Segmental and global amyloid burden and myocardial mechanics: a multimodality imaging comparison. (2026/05/01) ♡
- Procedural outcomes in patients undergoing non-cardiac surgery with cardiac variant amyloidosis. (2026/04/30) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Ophthalmological Manifestations of Hereditary Amyloidosis due to Transthyretin: A Systematic Review. (2026/04/30) ♡
- Structures of dye-bound transthyretin amyloid fibrils from abdominal fat biopsies. (2026/04/24) ♡
- Transferability of a US claims-based machine learning model for ATTRwt-CM identification: a retrospective evaluation in a German setting. (2026/04/24) ♡
- Gut-Heart Axis: Microbiome Involvement in Wild-Type Transthyretin Amyloidosis. (2026/04/23) ♡
- Cascade genetic screening in hereditary transthyretin amyloidosis: when early diagnosis becomes survival. (2026/04/16) ♡
- Quality of life and effectiveness of vutrisiran as a treatment for hereditary transthyretin amyloidosis. (2026/04/16) ♡
- Assessing the real-world safety of vutrisiran for transthyretin-mediated amyloidosis with polyneuropathy: Based on WHO-VigiAccess and FAERS databases. (2026/04/15) ♡
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