CORDIS Project
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This project aims to develop new chemical compounds that can increase the levels of frataxin in patients with Friedreich ataxia, a neurodegenerative disease. By preventing the degradation of this protein, the project seeks to improve the health outcomes of affected individuals.
Friedreich ataxia (FRDA) is a devastating neurodegenerative orphan disease that affects children and young adults, and has no approved therapy.
The disease progressively brings patients to severe disability and significantly reduces life expectancy.
The genetic defect underlying the disease causes FRDA patients to produce only low amounts of the mitochondrial protein frataxin, compared to normal subjects.
Low frataxin results in the accelerated death of peripheral sensory neurons, causing the di…
UNIVERSITA DEGLI STUDI DI ROMA TOR VERGATA
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