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Hereditary ATTR amyloidosis

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Search Period All yearsLast 12 monthsLast 5 years Topic All topicsGeneralNutrition and dietAlternative treatment methods Type of research Any type of researchOnly the strongest evidence (80)Meta-analysis (11)Treatment guideline (1)Phase 3 study (30)Randomized trial (23)Systematic review (15)Clinical trial (344)Review article (146)Early study (phase 1 or 2) (7)Case report (135)Laboratory or animal study (8)Commentary or editorial (72) Studies currently recruiting participants only (35)

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Publications and studies (1285)

• Angiographic Signatures of the Predominant Form of Familial Transthyretin Amyloidosis (Val30Met Mutation). (2018/08/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Integrating imaging modalities for diagnosing cardiac amyloidosis. (2018/08/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Myocardial Contraction Fraction by M-Mode Echocardiography Is Superior to Ejection Fraction in Predicting Mortality in Transthyretin Amyloidosis. (2018/08/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Gene-silencing technology gets first drug approval after 20-year wait. (2018/08/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. RNA-based therapies have their day. (2018/08/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Perforating palmar disease in TTR-related familial amyloid polyneuropathy. (2018/08/01) ♡
• Amyloid seeding of transthyretin by ex vivo cardiac fibrils and its inhibition. (2018/07/17) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Two cases of late onset familial amyloid polyneuropathy with a Glu61Lys transthyretin variant. (2018/07/15) ♡
• Transthyretin amyloid polyneuropathies mimicking a demyelinating polyneuropathy. (2018/07/10) ♡
• Unusual duplication mutation in a surface loop of human transthyretin leads to an aggressive drug-resistant amyloid disease. (2018/07/10) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Oligonucleotide Drugs for Transthyretin Amyloidosis. (2018/07/05) ♡
• 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. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. (2018/07/05) ♡
• 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. Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis. (2018/07/05) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Unmasking Early Wild-Type Transthyretin Amyloidosis Cardiomyopathy in a Patient With Refractory Atrial Fibrillation and Unremarkable Cardiac Imaging. (2018/07/01) ♡
• New Medications in the Treatment of Hereditary Transthyretin Amyloidosis. (2018/07/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Utility of Single-Photon Emission Computed Tomography/Computed Tomography Fusion Imaging With (99 m)Tc-Pyrophosphate Scintigraphy in the Assessment of Cardiac Transthyretin Amyloidosis. (2018/06/25) ♡
• Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis. (2018/06/09) ♡
• Understanding the Disease Course and Therapeutic Benefit of Tafamidis Across Real-World Studies of Hereditary Transthyretin Amyloidosis with Polyneuropathy: A Proof of Concept for Integrative Data Analytic Approaches. (2018/06/01) ♡
• 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. Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility. (2018/06/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Systemic angiopathy and axonopathy in hereditary transthyretin amyloidosis with Ala97Gly (p. Ala117Gly) mutation: a post-mortem analysis. (2018/06/01) ♡
• Cerebrospinal fluid and vitreous body exposure to orally administered tafamidis in hereditary ATTRV30M (p.TTRV50M) amyloidosis patients. (2018/06/01) ♡
• 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. Musculoskeletal Manifestations of Amyloidosis: A Focused Review. (2018/04/01) ♡
• Visualization of multiple organ amyloid involvement in systemic amyloidosis using (11)C-PiB PET imaging. (2018/03/01) ♡
• mtDNA copy number associated with age of onset in familial amyloid polyneuropathy. (2018/03/01) ♡
• Cardiac amyloidosis is associated with increased aortic stiffness. (2018/03/01) ♡
• 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. Tafamidis delays neurological progression comparably across Val30Met and non-Val30Met genotypes in transthyretin familial amyloid polyneuropathy. (2018/03/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. First nationwide survey on systemic wild-type ATTR amyloidosis in Japan. (2018/03/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Oculoleptomeningeal Amyloidosis Secondary to the Rare Transthyretin c.381T>G (p.Ile127Met) Mutation. (2018/03/01) ♡
• Regional Variation in Technetium Pyrophosphate Uptake in Transthyretin Cardiac Amyloidosis and Impact on Mortality. (2018/02/01) ♡
• Predictors of survival stratification in patients with wild-type cardiac amyloidosis. (2018/02/01) ♡
• Evidence that glial cells attenuate G47R transthyretin accumulation in the central nervous system. (2018/02/01) ♡
• Age-dependent cognitive dysfunction in untreated hereditary transthyretin amyloidosis. (2018/02/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Ocular Manifestations of Familial Transthyretin Amyloidosis. (2018/02/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Reply. (2018/02/01) ♡
• 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 amyloidosis: An update on pathophysiology, diagnosis, and treatment. (2018/01/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. RNA interference efficiently targets human leukemia driven by a fusion oncogene in vivo. (2018/01/01) ♡
• Clinical outcomes after preimplantation genetic diagnosis of patients with Corino de Andrade disease (familial amyloid polyneuropathy). (2018/01/01) ♡
• Frequency of and Prognostic Significance of Cardiac Involvement at Presentation in Hereditary Transthyretin-Derived Amyloidosis and the Value of N-Terminal Pro-B-Type Natriuretic Peptide. (2018/01/01) ♡
• Genetic and clinical characteristics of hereditary transthyretin amyloidosis in endemic and non-endemic areas: experience from a single-referral center in Japan. (2018/01/01) ♡
• Management of gastrointestinal complications in hereditary transthyretin amyloidosis: a single-center experience over 40 years. (2018/01/01) ♡
• [Transthyretin amyloidosis in a cohort of old and very old patients with chronic heart failure]. (2018/01/01) ♡
• Epidemiology of Transthyretin Familial Amyloid Polyneuropathy in Portugal: A Nationwide Study. (2018/01/01) ♡
• 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. Recent Advances in Oligonucleotide-Based Therapy for Transthyretin Amyloidosis: Clinical Impact and Future Prospects. (2018/01/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects (2018-09-21) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) (2018-07-18) ♡
• Transthyretin familial amyloid polyneuropathy (TTR-FAP): Parameters for early diagnosis. (2017/12/19) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. PET-CT evaluation of amyloid systemic involvement with [(18)F]-florbetaben in patient with proved cardiac amyloidosis: a case report. (2017/12/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Successful use of palliative inotrope therapy in end-stage cardiac ATTR amyloidosis. (2017/12/01) ♡
• Hip and knee arthroplasty are common among patients with transthyretin cardiac amyloidosis, occurring years before cardiac amyloid diagnosis: can we identify affected patients earlier? (2017/12/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety and efficacy of a TTR specific antisense oligonucleotide in patients with transthyretin amyloid cardiomyopathy. (2017/12/01) ♡

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