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    Friedreich Ataxia
    Home Our Science Friedreich's ataxia
  • What is it?

    Friedreich's Ataxia (FRDA) is a rare genetic disease characterized by loss of coordination and muscle strength resulting from the degeneration of nerve tissue in the spinal cord and damage of the peripheral nerves that control muscle movement. Symptoms range from the inability to coordinate movements to gait instability with imbalance, muscle weakness and tremors. Patients lose their ability to stand, sit and walk within 10 – 15 years of onset. FRDA reduces life expectancy and becomes fatal, mainly due to cardiac failure. Friedreich's Ataxia is caused by a genetic defect which affects male and female children and is inherited as a recessive trait. Deficient expression of the mitochondrial protein, frataxin, is the primary cause of FRDA, which leads to adverse alterations in whole cell and mitochondrial iron metabolism causing iron accumulation, mitochondrial dysfunction and oxidative damage. Therefore, the maintenance of mitochondrial homeostasis is crucial in the progression of neurodegenerative conditions, particularly in FRDA. The prevalence of FRDA is approximately 1 in 40,000 people. There is currently no known cure available and patients manage their disease with symptomatic treatment only.

  • Mind

    Orphan neurodegenerative mitochondrial disease

    DNA

    GAA repeats in the frataxin gene

    Baby

    Prevalence of 1/40,000

    Wheelchair

    Reduced frataxin levels leading to mitochondrial dysfunction

    People

    Main symptoms: Ataxia, Cardiomyopathy Diabetes mellitus 

    Brain

    Early-intermediate onset between 5 and 24 years of age

  • Our Programs
    Delivering innovative treatments
    Our wholly-owned lead asset, leriglitazone, is currently in clinical development for the treatment of a range of orphan CNS disorders with high unmet medical need.
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    by eMascaró