Safety and Efficacy of Interferon γ in Friedreich's Ataxia

Marinela Vavla MD PhD Maria Grazia D'Angelo MD PhD Filippo Arrigoni MD Nicola Toschi PhD Denis Peruzzo PhD Sandra Gandossini MD Annamaria Russo MD Eleonora Diella Stefania Tirelli PhD Roberto Salati MD Paolo Scarpazza MD Riccardo Luffarelli PhD Silvia Fortuni PhD Alessandra Rufini PhD Ivano Condò PhD Roberto Testi MD Andrea Martinuzzi MD PhD; Mov Disord. 2020 doi:10.1002/mds.27979

A relevant indication that emerges from the study, the previously studies, and from the numerous FRDA patients using IFNγ off label is the possible presence of nonresponders. A randomized withdrawal design is therefore best suited to increase the power of future clinical trials to definitively assess the efficacy of IFNγ treatment in FRDA


Safety and Efficacy of Interferon γ in Friedreich's Ataxia