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Rare and hereditary disorders
Hereditary ATTR amyloidosis
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Publications and studies (1285)
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Transthyretin Cardiac Amyloidosis in Australia and New Zealand-A Multi-Site Snapshot for 2022. (2025/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Quantitative muscle MRI combined with AI-based segmentation as a follow-up biomarker for ATTRv patients: A longitudinal pilot study. (2025/01/01) ♡
- Real-life experience with disease-modifying drugs in hereditary transthyretin amyloid polyneuropathy: A clinical and electrophysiological appraisal. (2025/01/01) ♡
- Recovery of right ventricular function in patients with transthyretin cardiac amyloidosis after one-year tafamidis administration. (2025/01/01) ♡
- Misfolding of transthyretin in vivo is controlled by the redox environment and macromolecular crowding. (2025/01/01) ♡
- 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. Estimating Meaningful Differences in Measures of Neuropathic Impairment, Health-Related Quality of Life, and Nutritional Status in Patients With Hereditary Transthyretin Amyloidosis. (2025/01/01) ♡
- Meta-analysisiAll studies on one question combined and calculated together. This is the strongest form of research that exists: a single loose study can be coincidence, dozens together much less so. The label says something about the design, not about the outcome — which can also be that something does NOT work. Neurological efficacy and safety of RNA therapeutics in hereditary transthyretin amyloidosis: a systematic review and meta-analysis of randomized controlled trials. (2025/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Amyloidosis TTR Flow Reserve Evaluation (2025-12-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Multimodality Cardiac Imaging for Disease Progression in ATTR-CM (2025-12-09) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (2025-12-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Cardiac Amyloidosis Discovery Trial (2025-12-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Research Study to Look at Long-term Treatment With a Medicine Called NNC6019-0001 for People Who Have Heart Failure Due to Transthyretin Amyloidosis (2025-11-25) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Imaging Cardiac Amyloidosis: A Pilot Study Using F-18 Florbetapir Positron Emission Tomography (2025-11-20) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Screening for Cardiac Amyloidosis With Nuclear Imaging for Minority Populations (2025-11-19) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. [18F]FT8 PET Imaging in Immunoglobulin Light Chain Amyloidosis (2025-11-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Ultrasound Therapy In Cardiac Amyloidosis (2025-11-14) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. US Benchmarking Clinical Study (2025-10-20) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants (2025-10-14) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluation of a Claims-based Algorithm for the Identification of Transthyretin-mediated Amyloidosis (ATTR) Amyloidosis in Medical Records (2025-09-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Transthyretin Amyloidosis Cardiomyopathy in Patients With HFpEF in Russia (2025-09-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Hybrid Florbetaben PET/MRI for Imaging of Cardiac Amyloidosis (2025-09-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Cardiac Amyloid Deposits and Heart Dysfunction in Alzheimer's Disease (2025-09-04) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Essen Amyloidosis Registry (2025-08-21) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Pan-Amyloid PET/CT in Various Amyloid-Related Disease (2025-08-19) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. TEGSEDI Pregnancy Surveillance Program (2025-08-19) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Prevalence of wtATTR-CM After Carpal Tunnel Release Surgery (2025-08-15) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Phenotyping and Characterization of wtATTR-CM (TRACE 1) (2025-08-15) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Multimodal Radiomics Model (18F-FAPI PET/CT + CMR) for AL Cardiac Amyloidosis Prognosis (2025-08-05) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Parametric Cardiac 18F-flutemetamol PET Imaging in ATTR Cardiomyopathy (2025-08-03) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy (2025-07-30) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis. (2025-07-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR (2025-07-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects (2025-06-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Exercise in Hereditary ATTR (ATTRv) Amyloidosis (2025-06-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Genistein in trAnSthyretin recePtor Amyloid caRdiomyopathy (2025-06-15) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Longitudinal Quantitative Neuromuscular MRI in Neuropathic Patients (2025-05-31) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. hATTR Polyneuropathy in Russia (2025-05-25) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Swiss Cardiac Amyloidosis REgistry (Swiss-CARE) (2025-05-06) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hATTR-PN (2025-04-10) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An Interventional Pilot Study to Investigate the Feasibility and Acceptance of a Structured Psychological Support Program for Patients, Caregivers, and Presymptomatic Carriers in Hereditary Transthyretin Amyloidosis With Cardiomyopathy (2025-04-06) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Description of a Retrospective Cohort of Patients With Transthyretin Cardiac Amyloidosis (ATTR-CA) in Internal Medicine (2025-04-02) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. International Cardiac Amyloidosis Registry (2025-03-25) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Echocardiographic Characterization of Cardiac Amyloidosis (2025-03-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Exercise Training in Transthyretin Cardiac Amyloidosis (2025-03-18) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Physiologic Assessment of Microvascular Function in Patients With Cardiac Amyloidosis (2025-03-14) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Open-Label Study of AG10 in Patients with Cardiomyopathy (2025-03-13) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Role of Occult Cardiac Amyloid in the Elderly With Aortic Stenosis. (2025-03-11) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Observational Epidemiological Study of Patients Suffering From Systemic Amyloidosis (2025-03-10) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy (2025-02-14) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Screening for Amyloidosis Before Aortic Valve Elective Replacement (2025-02-12) ♡
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