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Fabry disease
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Studies (254)
- 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) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease (2024-10-08) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Studying Lipids as Potential Biomarkers in Patients With Fabry Disease (2024-10-01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Multimodal and Multidisciplinary Approach to Optimize Diagnostic, Prognostic, and Therapeutic Management of Patients with Non-ischemic Cardiomyopathies and Arrhythmogenic-inflammatory Phenotypes: a Multicenter, Observational, Retrospective and Prospective Registry Study. (2024-09-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients (2024-09-19) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Screening of Fabry Disease in Patients With Left Ventricular Hypertrophy Detected in Echocardiography (2024-08-20) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (2024-08-09) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients (2024-07-31) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease (2024-07-30) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease (2024-07-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Molecular Imaging in Fabry Disease of the Heart (2024-07-22) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease (2024-07-09) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of Replagal in Treatment-naïve Adults With Fabry Disease (2024-06-17) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score (2024-06-12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluation of a Screening Strategy of Fabry Disease in Patient With Renal Biopsy (2024-06-11) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences (2024-05-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat (2024-05-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease (2024-04-17) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study of the Effects of Fabrazyme Treatment on Lactation and Infants (2024-04-10) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease (2024-04-08) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients (2024-04-03) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Real World Evidence Study of Danish Fabry Patients (2024-03-12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE (HOPE Fabry) (2024-03-06) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Canadian Fabry Disease Initiative (CFDI) National Registry (2024-02-15) ♡
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