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Friedreich's ataxia is an autosomal‐recessive cerebellar ataxia caused by mutation of the frataxin gene, resulting in decreased frataxin expression, mitochondrial dysfunction, and oxidative stress. Currently, no treatment is available for… Click to show full abstract
Friedreich's ataxia is an autosomal‐recessive cerebellar ataxia caused by mutation of the frataxin gene, resulting in decreased frataxin expression, mitochondrial dysfunction, and oxidative stress. Currently, no treatment is available for Friedreich's ataxia patients. Given that levels of residual frataxin critically affect disease severity, the main goal of a specific therapy for Friedreich's ataxia is to increase frataxin levels.
Keywords:
drug repositioning;
friedreich ataxia;
frataxin;
ataxia;
repositioning screening;
screening identifies
Journal Title: Movement Disorders
Year Published: 2019
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Journal Title: Movement Disorders
Year Published: 2019
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!