New developments in pharmacotherapy for Friedreich ataxia article

Current and emerging therapies for Friedrich’s Ataxia (FA) focus on reversing the deleterious effects of such deficiency including mitochondrial augmentation and increasing frataxin levels, providing the possibility of treatment options for this physiologically complex, multisystem disorder. In this review article, the authors discuss the current and prior in vivo and...

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Gene therapy abroad

Neurocrine Biosciences and Voyager Therapeutic announced the formation of a strategic collaboration focused on the development and commercialization of Voyager’s gene therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, as well as rights to two programs to be determined. This collaboration combines Neurocrine Biosciences’ expertise in neuroscience,...

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