Nomlabofusp (CTI-1601) at high dose safely raises frataxin levels
Use of the investigational therapy CTI-1601, now known as nomlabofusp, was associated with dose-dependent increases in levels of frataxin — the protein whose lack causes Friedreich’s ataxia (FA) — in skin and buccal cells of adult patients in a clinical trial. Treatment tolerability also was reported in top-line…