Fabry Disease
📧 Sign up to get email alerts when something new happens for Fabry Disease.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS research · 2025-11-04Clinical Trial Update: A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease [Phase 1, Phase 2]
A phase 1/2 proof-of-concept trial is assessing safety and tolerability of HM15421/GC1134A in patients with Fabry disease; study is currentl…
-
CLINICAL_TRIALS research · 2025-10-23Clinical Trial Update: Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease [Phase 1, Phase 2]
This is a clinical trial update describing a Phase 1/2 study of AMT-191 in classic Fabry disease, assessing safety, PK/PD, and exploratory e…
-
CLINICAL_TRIALS research · 2025-09-12Clinical Trial Update: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
This clinical trial update describes ScreenPlus, a comprehensive, multi-disorder newborn screening program that is currently enrolling by in…
-
CLINICAL_TRIALS research · 2025-08-22Clinical Trial Update: Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
ClinicalTrials.gov listing for a study on the natural history and structural-functional relationships in Fabry disease with renal involvemen…
-
CLINICAL_TRIALS research · 2025-07-08Clinical Trial Update: A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
A global prospective observational study sponsored by Amicus Therapeutics, evaluating pregnancy and breastfeeding outcomes in women with Fab…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS research · 2025-07-03Clinical Trial Update: Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
A physician-initiated expanded access request for migalastat in individual Fabry disease patients is being tracked, with the program listed …
-
CLINICAL_TRIALS research · 2025-05-14Clinical Trial Update: Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease [NA]
Clinical trial update on arrhythmia burden and risk of sudden cardiac death and stroke in patients with Fabry disease; the study is currentl…
-
CLINICAL_TRIALS research · 2025-05-04Clinical Trial Update: Aortic Dimensions in Patients With Fabry Disease
A clinical trial evaluating aortic dimensions in patients with Fabry disease is currently not yet recruiting, sponsored by University Hospit…
-
CLINICAL_TRIALS research · 2025-04-08Clinical Trial Update: the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease
This clinical trial investigates the role of cardiac inflammation, endothelial dysfunction, and fibrosis in Fabry disease and is currently r…
-
CLINICAL_TRIALS research · 2025-04-06Clinical Trial Update: Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease
A clinical trial is recruiting to study fibrosis, inflammation, and oxygenation in renal tissue for Fabry disease with chronic kidney diseas…
-
CLINICAL_TRIALS research · 2025-03-30Clinical Trial Update: German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
This clinical trial update describes an observational multicenter study in Germany evaluating patients with Fabry disease who are receiving …
-
CLINICAL_TRIALS research · 2025-03-26Clinical Trial Update: CVI Alterations in FD: a Prospective, Multicenter, Observational Cohort Study
A prospective, multicenter observational cohort study evaluating cardiovascular alterations in Fabry disease with cardiac involvement.
-
CLINICAL_TRIALS research · 2025-02-21Clinical Trial Update: 4D-310 in Adults With Fabry Disease and Cardiac Involvement [Phase 1, Phase 2]
An ongoing Phase 1/2 clinical trial evaluating 4D-310 in adults with Fabry disease and cardiac involvement is currently recruiting.
-
CLINICAL_TRIALS research · 2025-02-11Clinical Trial Update: The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease [Phase 1, Phase 2]
This document reports a Phase 1/2 clinical trial evaluating intravenous EXG110 in patients with Fabry disease, conducted by Guangzhou Jiayin…
-
CLINICAL_TRIALS research · 2025-02-07Clinical Trial Update: Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation
This clinical trial update describes a study at IRCCS Azienda Ospedaliero-Universitaria di Bologna that aims to characterize patients with v…
-
CLINICAL_TRIALS research · 2025-02-04Clinical Trial Update: Italian Anderson Fabry Disease Cardiovascular Registry
This clinical trial registry entry describes the Italian Anderson Fabry Disease Cardiovascular Registry, a recruiting project to collect car…
-
CLINICAL_TRIALS research · 2025-01-06Clinical Trial Update: Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational Study
A prospective observational study at Technical University of Munich aims to characterize the retinal microvasculature in Fabry disease patie…
-
CLINICAL_TRIALS research · 2024-07-30Clinical Trial Update: Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease [Phase 2]
Clinical trial update for a Phase 2 study of AL01211 in treatment-naive males with classic Fabry disease; the study is currently recruiting …
-
CLINICAL_TRIALS research · 2024-07-29Clinical Trial Update: Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease [Phase 3]
ISU Abxis Co., Ltd. is conducting a Phase 3 trial to evaluate the safety and efficacy of Fabagal (agalsidase beta) for Fabry disease, with p…
-
CLINICAL_TRIALS research · 2024-07-22Clinical Trial Update: Molecular Imaging in Fabry Disease of the Heart
A University of Cambridge-led clinical trial is recruiting to study molecular imaging in Fabry disease of the heart (cardiac variant).
-
CLINICAL_TRIALS research · 2024-07-09Clinical Trial Update: Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease [EARLY_Phase 1]
A Phase 1 pediatric trial is evaluating a gene therapy drug for Fabry disease, focusing on safety, tolerability, and efficacy. The study is …
-
CLINICAL_TRIALS research · 2024-06-12Clinical Trial Update: Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score [NA]
This clinical trial update examines the prevalence of Fabry disease in high-risk patients with left ventricular hypertrophy and the implemen…
-
CLINICAL_TRIALS research · 2024-06-11Clinical Trial Update: Evaluation of a Screening Strategy of Fabry Disease in Patient With Renal Biopsy [NA]
Clinical trial evaluating a screening strategy for Fabry disease in patients undergoing renal biopsy; recruitment is ongoing at University H…
-
CLINICAL_TRIALS research · 2024-05-23Clinical Trial Update: Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
A completed clinical trial update evaluating treatment-related benefits and patient satisfaction in Fabry disease. It provides insights into…
-
CLINICAL_TRIALS research · 2018-07-23Clinical Trial Update: Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients
An epidemiological clinical trial update studying Fabry disease among young stroke patients in Taiwan, sponsored by Chiayi Christian Hospita…