About This Study
Fabry disease is a rare genetic condition where the body cannot break down certain fats, causing them to build up and damage organs like the heart, kidneys, and nerves. The purpose of this study is to learn more about the medicine migalastat for this condition. Our researchers want to see if it is safe and helpful for children.
What Activities Are a Part of this Study?
- Informed Consent Review
- Use of Study Treatments or Placebo
- Use of Study Health Plans
- After Study Follow-Up Activities
- Review of Medical History
- Physical Examinations
- Phone Calls and/or Emails
- Study Visits and Consultations
- Surveys/Questionnaires
- Laboratory and Imaging Tests
- Specimen Sample Collection
Age & Gender
- 2 - 12 years old
- Female
- Male
Language
- This study enrolls English speakers only
Additional Study Information
- Compensation Offered
- Reimbursement offered
Study Locations
- Greater Charlotte, NC
Study Topics
Children, Infants, Teen Health Genetic Disorders Rare Disease