Question 17344 — diseases

Summary

Mr. Julien Aubert draws the attention of the Minister of Solidarity and Health to Friedreich's ataxia. This rare and incurable hereditary disease is characterized by neurodegenerative damage, heart damage and an increased risk of diabetes. Currently, this disease has no treatment. However, recent experiments on mice, carried out by the Institute of Genetics and Molecular and Cellular Biology (IBGMC) of Strasbourg, demonstrated the effectiveness of gene therapy on the neurodegenerative damage associated with Friedreich's ataxia. This therapy is advanced and benefits from several years of research in the preclinical phase but is unfortunately not yet accessible to patients to date. Friedreich's ataxia being a neurodegenerative disease inducing deterioration from day to day, he asked him what the Government, in terms of experimentation, of gene therapy aimed at patients who would like to be able to benefit from it without delay.

Machine translation from French. The official text remains authoritative.

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