The PROFA study is an international, multi-centric observational and validation study to assess the patient-reported, psychosocial and economic outcomes of patients with Friedreich Ataxia (FA). Eligible patients will be recruited from six study centers in Germany, Austria and France. Patients will complete a baseline assessment via face-to-face interviews at the study centers and multiple momentary follow-up assessments via a mobile-health app at home daily to monthly for six months. Study results will gain essential and in-depth insights into the daily life of patients with FA.
FA confirmed by molecular genetic testing
Ataxia severity of ≤30 points according to the Scale of the Assessment and Rating of Ataxia (SARA)
Access to a smartphone or tablet and able to operate the device
Older than 12 years
Lack of ability to give consent
Ataxia severity >30 according to the Scale of the Assessment and Rating of Ataxia (SARA)
German Center for Neurodegenerative Diseases (DZNE)
Lead sponsor
McMaster University
Collaborator
Sorbonne University
Collaborator