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European Consortium for Translational Studies in Friedreich’s Ataxia (EFACTS) Patient Registry

The main criteria for participation

The EFACTS registry is recruiting, in Europe and particularly in France, 1,200 people of all ages, who have a genetic diagnosis of Friedreich’s ataxia, or conversely whose absence of Friedreich’s ataxia is genetically confirmed.

The objective

The registry aims to establish the natural history of Friedreich’s ataxia, to correlate clinical manifestations of the disease with proteomic data, to accelerate the identification and recruitment of individuals for future clinical trials, and to identify biomarkers of changes in the natural history of the disease that could be used both in management and in clinical trials.

In practice

During an annual visit to the research center, the participant undergoes a battery of tests to assess their clinical symptoms, motor and cognitive functions, and quality of life. They also provide biological samples (blood, urine, etc.).

Where are we?

Recruitment is underway at the Brain Institute in Paris and has not yet begun at Hautepierre Hospital in Strasbourg.

Contact :

Elodie Petit (Paris Brain Institute – ICM)

elodie.petit@icm-institute.org

Tel. : 01 57 27 45 31

Mathieu Anheim (Hautepierre Hospital, Neurology Department, Strasbourg)

mathieu.anheim@chru-strasbourg.fr

Tel. : 03 88 12 87 11

See the full description of the study