Summary

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• This summary is based on the review of one systematic review/meta-analysis. ^[1]
• The effectiveness of omaveloxolone was evaluated in three studies; however, specific findings regarding its impact on the International Cooperative Ataxia Rating Scale (ICARS), Friedreich Ataxia Rating Scale (FARS), Scale for Assessment and Rating of Ataxia (SARA), and Activities of Daily Living scale (ADL) were not explicitly detailed, implying inconclusive results or possible deterioration in Friedreich's ataxia (FA) severity.
• The systematic review included patients aged 8 to 73 years with disease durations ranging from 4.7 to 19 years and mean GAA1 and GAA2 allele repeat lengths from 350 to 930 and 620 to 987 nucleotides, respectively, though no significant differences in treatment effectiveness among different population types or subgroups were reported.
• Other therapeutic interventions, such as idebenone and recombinant erythropoietin, similarly demonstrated inconclusive results or deterioration in effectiveness, reflecting a significant unmet need for effective therapies in Friedreich ataxia.
• The therapeutic interventions studied, including omaveloxolone, were generally well-tolerated and safe, with serious adverse events reported being atrial fibrillation (1 case), craniocerebral injury (1 case), and ventricular tachycardia (1 case).
• The safety profile of omaveloxolone was not specifically detailed; however, it was grouped with other treatments that showed no significant safety concerns or adverse effects beyond those expected in the patient population.
• There is no population types or subgroups information available in the reviewed documents.