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Fabry disease

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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 researchHighest evidence only (55)Meta-analysis (9)Treatment guideline (2)Phase 3 study (19)Systematic review (19)Randomized trial (6)Clinical trial (301)Review article (188)Early study (phase 1 or 2) (4)Case report (159)Laboratory or animal study (24)Commentary or editorial (52)Retracted publication (2) Only studies currently seeking participants (25)

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Publications and studies (1331)

• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease (2025-11-04) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease (2025-10-23) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme (2025-10-21) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion (2025-10-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies (2025-10-02) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of Replagal in Children and Adults With Fabry Disease in India (2025-09-19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. China Post-marketing Surveillance (PMS) Study of Fabrazyme® (2025-09-11) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study (2025-08-22) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Anderson Fabry Disease - the Power of Information. (2025-08-22) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding (2025-07-08) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease (2025-07-03) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing (2025-06-19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Long-Term Follow-up of Subjects Who Were Treated With ST-920 (2025-05-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Screening for Fabry Disease in Renal Transplantation (2025-05-16) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease (2025-05-14) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Aortic Dimensions in Patients With Fabry Disease (2025-05-04) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease (2025-04-08) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease (2025-04-06) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. CVI Alterations in FD: a Prospective, Multicenter, Observational Cohort Study (2025-03-26) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Autoimmune and Inflammatory Response Biomarkers in Fabry Disease (2025-03-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. 4D-310 in Adults With Fabry Disease and Cardiac Involvement (2025-02-21) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Co-designed Physical Activity Intervention in Fabry Disease (2025-02-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease (2025-02-11) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation (2025-02-07) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Italian Anderson Fabry Disease Cardiovascular Registry (2025-02-04) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Diagnostic Role of Renal Biopsy in Patients With Fabry Disease (2025-01-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational Study (2025-01-06) ♡
• Role of Genetic Testing in Diagnosis and Prognosis Prediction in Hypertrophic Cardiomyopathy in Korea. (2024/12/30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Impact of enzyme replacement therapy on clinical manifestations in females with Fabry disease. (2024/12/27) ♡
• Role of Longitudinal Strain in the Evaluation of Contractile Dysfunction in Japanese Fabry Disease Patients. (2024/12/25) ♡
• 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 case of Fabry disease with cardiac injury as the main clinical manifestation]. (2024/12/24) ♡
• UPLC-MS/MS High-Risk Screening for Sphingolipidoses Using Dried Urine Spots. (2024/12/17) ♡
• Health care costs of home care enzyme replacement therapy for patients with lysosomal storage diseases in Germany. (2024/12/16) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Echocardiography-guided percutaneous intramyocardial septal radiofrequency ablation procedure for the treatment of Fabry disease: a case report. (2024/12/14) ♡
• Targeted nanoliposomes to improve enzyme replacement therapy of Fabry disease. (2024/12/13) ♡
• 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. Unveiling the Future of Cardiac Care: A Review of Gene Therapy in Cardiomyopathies. (2024/12/06) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Rescue from sudden ventricular tachycardia and fibrillation using wearable cardioverter-defibrillator in male late-onset Fabry disease patient. (2024/12/06) ♡
• Pharmacokinetic evaluation of single-dose migalastat in non-Fabry disease subjects with ESRD receiving dialysis treatment, and use of modeling to select dose regimens in Fabry disease subjects with ESRD receiving dialysis treatment. (2024/12/05) ♡
• [Clinical features and quality of life in patients with Fabry disease]. (2024/12/03) ♡
• 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. The contribution of the sphingosine 1-phosphate signaling pathway to chronic kidney diseases: recent findings and new perspectives. (2024/12/01) ♡
• Characterization of Fabry disease-associated lyso-Gb(3) on mouse colonic ion transport and motility. (2024/12/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Identification of c.146G > A mutation in a Fabry patient and its correction by customized Cas9 base editors in vitro. (2024/12/01) ♡
• Genotype-Phenotype Spectrum of 52 Mexican Patients With Fabry Disease: A Novel GLA Variant With Atypical Phenotype. (2024/12/01) ♡
• Biomarkers Reflecting the Severity of Bronchial Asthma in Children. (2024/11/29) ♡
• Long-read sequencing enables comprehensive molecular genetic diagnosis of Fabry disease. (2024/11/28) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Glycosphingolipids and their impact on platelet activity in a murine model of Fabry disease. (2024/11/27) ♡
• Overcoming Resistance in Anderson-Fabry Disease: Current Therapeutic Challenges and Future Perspectives. (2024/11/27) ♡
• Liver Lesions in Estuarine Dolphins in the Indian River Lagoon, Florida: Does Microcystin Play a Role? (2024/11/27) ♡
• Fabry Disease Podocytes Reveal Ferroptosis as a Potential Regulator of Cell Pathology. (2024/11/23) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Case Report: Diagnostic challenges in Fabry disease: misinterpreted obstructive hypertrophic cardiomyopathy and the role of enzyme replacement therapy. (2024/11/22) ♡

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