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Friedreich's ataxia (FRDA) is a progressive disease affecting multiple organs that is caused by systemic insufficiency of the mitochondrial protein frataxin. Current therapeutic strategies aim to elevate frataxin levels and/or… Click to show full abstract
Friedreich's ataxia (FRDA) is a progressive disease affecting multiple organs that is caused by systemic insufficiency of the mitochondrial protein frataxin. Current therapeutic strategies aim to elevate frataxin levels and/or alleviate the consequences of frataxin deficiency. Recent significant advances in the FRDA therapeutic pipeline are bringing patients closer to a cure.
Keywords:
friedreich ataxia;
therapeutic prospects;
prospects friedreich
Journal Title: Trends in pharmacological sciences
Year Published: 2019
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Journal Title: Trends in pharmacological sciences
Year Published: 2019
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!