Friedreich’s ataxia is a rare nervous system degenerative disease that leads to uncoordinated muscle movement, impaired walking, speech, and swallowing, poor balance, and shortened lifespan. To treat Friedreich’s ataxia in patients aged 16 years and above , the USFDA has recently approved omaveloxolone . The approval was granted based on the findings of a 48-week, randomized, double-blind study (Study 1) and an open-label extension of the study.
##Clinical trial## In Study 1, 103 Friedreich’s ataxia patients, aged between 16 and 40 years, received 150 mg of omaveloxolone (n = 51) or placebo (n = 52). Upper and lower limb coordination, upright stability, and swallowing and speech were measured using a modified Friedreich’s Ataxia Rating Scale (mFARS) at week 48 . Patients who received omaveloxolone achieved significantly lower mFARS scores due to reduced impairment compared to the…
