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Rare and hereditary disorders
Hereditary ATTR amyloidosis
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Publications and studies (1285)
- Standard heart failure medication in cardiac transthyretin amyloidosis: useful or harmful? (2017/03/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Efficiency of siRNA for removal of transthyretin V30M in a TTR leptomeningeal animal model. (2017/03/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Transthyretin amyloidosis: a little history of hereditary amyloidosis. (2017/03/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. An aggressive form of transthyretin amyloidosis. (2017/03/01) ♡
- Occurrence factors and clinical picture of iatrogenic transthyretin amyloidosis after domino liver transplantation. (2017/03/01) ♡
- The demographic, genetic, and clinical characteristics of Brazilian subjects enrolled in the Transthyretin Amyloidosis Outcomes Survey. (2017/03/01) ♡
- The ever-growing understanding of transthyretin amyloidosis nephropathy. (2017/03/01) ♡
- The demographic, genetic, and clinical characteristics of Latin American subjects enrolled in the Transthyretin Amyloidosis Outcomes Survey. (2017/03/01) ♡
- Clinicopathological and biochemical findings of late-onset hereditary transthyretin amyloidosis 16 years after liver transplantation: an autopsy case study. (2017/03/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Transthyretin-related hereditary amyloidosis in an Argentinian family with TTR Tyr114Cys mutation. (2017/03/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. De novo hereditary (familial) amyloid polyneuropathy (FAP) in a FAP liver recipient. (2017/03/01) ♡
- Disruption of blood-nerve barriers in hereditary transthyretin (ATTR) amyloidosis. (2017/03/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Hereditary transthyretin amyloidosis associated with a transthyretin variant Thr59Arg. (2017/03/01) ♡
- Refine penetrance estimates in the main pathogenic variants of transthyretin hereditary (familial) amyloid polyneuropathy (TTR-FAP) using a new non-parametric approach (NPSE). (2017/03/01) ♡
- End-stage renal failure due to transthyretin amyloidosis after liver transplantation: outcomes in 19 registry cases. (2017/03/01) ♡
- 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 late-onset case of hereditary transthyretin amyloidosis with a novel compound heterozygous mutation. (2017/03/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Transthyretin (ATTR) amyloidosis nephropathy: lessons from a TTR stabilizer molecule. (2017/03/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Doxycycline-tauroursodeoxycholic acid treatment: effects in the heart of a transthyretin V30M transgenic mouse model. (2017/03/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Clinical relevance of pulmonary amyloidosis: an analysis of 76 autopsy-derived cases. (2017/02/23) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. [Treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a case report]. (2017/01/01) ♡
- Subjects at Risk for Genetic Late-Onset Neurological Diseases: Objective Knowledge. (2017/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Frequency of Cardiac Amyloidosis in the Caribbean's. (TEAM Amylose) (2017-10-27) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Prospective Identification of Cardiac Amyloidosis by Cardiac Magnetic Resonance Imaging (2017-10-26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Thalidomide/Dexamethasone Treatment And PET Evaluation In Organ Involvemenet of Cardiac Amyloidosis (2017-10-26) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Radioisotope Scintigraphy to Establish Incidence of Cardiac Amyloidosis Among Patients With Otherwise Unexplained Cardiac Disease (2017-04-05) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Effect of Diflunisal on Familial Amyloidosis (2017-03-17) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study of SOM0226 in Familial Amyloid Polyneuropathy (2016-11-30) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart (2016-08-10) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Efficacy Study of Doxycycline/UrsoDeoxyCholicAcid on Disease Progression in ATTR Amyloidosis (2016-05-12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety, Efficacy and Pharmacokinetics of Doxycycline Plus Tauroursodeoxycholic Acid in Transthyretin Amyloidosis (2016-02-25) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Effect of Diflunisal on Familial Transthyretin Amyloidosis (2015-08-24) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Effects of Fx-1006A on Transthyretin Stabilization and Clinical Outcome Measures in Patients With Non-V30M Transthyretin Amyloidosis (2013-01-18) ♡
- 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. Experimental inhibition of fibrillogenesis and neurotoxicity by amyloid-beta (Aβ) and other disease-related peptides/proteins by plant extracts and herbal compounds. (2012/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis (2012-12-17) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy (2012-12-17) ♡
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