申请人:Fratagene Therapeutics S.R.L.
公开号:US10442779B2
公开(公告)日:2019-10-15
The invention provides compositions and methods useful for the treatment of Friedreich's ataxia. In some embodiments, the invention provides compositions and methods useful for inhibiting the ubiquitination of frataxin.
本发明提供了用于治疗弗里德里希共济失调的组合物和方法。在某些实施方案中,本发明提供了抑制 frataxin 泛素化的组合物和方法。