Amicus Therapeutics

NCT00351156

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

NCT00515398

A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

NCT03158662

Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa

NCT03135197

German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.

NCT02363153

Diet and Exercise in Pompe Disease

NCT03347253

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

NCT03838237

Effect of Migalastat on Cardiac Involvement in Fabry Disease

NCT04043273

Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences

NCT04184986

Screening of Fabry Disease in Patients With GI Symptoms

NCT04129905

Assessment of the Relations Between Endothelial and Venous Dysfunctions and Left Ventricular Obstruction in Genetic Hypertrophic Cardiomyopathies

NCT04281537

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

NCT04602364

French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat

NCT04804566

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

NCT06303466

Real World Evidence Study of Danish Fabry Patients

NCT06121011

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT04252066

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

NCT06906367

A Study of Patients With Fabry Disease (US Specific)