Omaveloxolone for the Treatment of Friedreich’s Ataxia
نویسندگان
چکیده
Friedrich’s ataxia (FRDA), a neurodevelopmental and progressive neurodegenerative disease, is the most common inherited form of ataxia. Omaveloxolone was approved by US Food Drugs Administration in early 2023, making it first treatment available to patients with FRDA. This approval made possible combining compelling cellular mechanism strong clinical evidence provided through MOXIe study, multipart trial evaluating efficacy omaveloxolone review discusses underlying pathology proposed The study presented detail, including discussion challenges faced trials FRDA, rare diseases more broadly. Finally, other therapies under investigation are reviewed briefly.
منابع مشابه
Dysarthria in Friedreichs Ataxia: A Perceptual Analysis
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ژورنال
عنوان ژورنال: US neurology
سال: 2023
ISSN: ['1758-4000', '1758-4019']
DOI: https://doi.org/10.17925/usn.2023.19.2.2