Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
Friedreich's Ataxia Research Alliance
Summary
This project is a global, multicenter, prospective, longitudinal, observational natural history study that can be used to understand the disease progression and support the development of safe and effective drugs and biological products for Friedreich ataxia.
Description
The UNIFAI Study: Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study is a global research study designed to provide a deep and evolving understanding of the natural history of this rare and debilitating genetic disorder as well as inform clinical trial design and implementation. Friedreich's Ataxia is a rare, inherited, multi-system condition characterized by progressive neurological and cardiac symptoms. It is caused by mutations in the FXN (frataxin) gene. The UNIFAI study is a multicenter, prospective, longitudinal, observational study, which means that all data ab…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- Yes
Inclusion Criteria: 1. Both males and females of any age 2. Individuals with Friedreich ataxia (FA): Participants that meet genetically confirmed diagnosis of Friedreich ataxia 3. Written informed consent provided 1. Informed consent must be obtained for all participants 2. For underage participants, they and the parent/ legally authorized representative have to sign the informed consent form, child assent (if applicable) 3. Persons who are not legally competent require the informed consent of their legally authorized representative Exclusion Criteria: 4. Unable or unwilling to…
Locations (34)
- UCLA Ataxia CenterLos Angeles, California
- University of ColoradoDenver, Colorado
- University of Florida - NeurologyGainesville, Florida
- USF Ataxia Research CenterTampa, Florida
- Emory University Hospital - NeurologyAtlanta, Georgia
- University of Iowa, Stead Family Children's HospitalIowa City, Iowa