Omaveloxolone: an activator of Nrf2 for the treatment of Friedreich ataxia.
Expert Opin Investig Drugs
; 32(1): 5-16, 2023 Jan.
Article
en En
| MEDLINE
| ID: mdl-36708320
ABSTRACT
INTRODUCTION:
Friedreich ataxia (FRDA) is a rare autosomal recessive degenerative disorder characterized by ataxia, dysarthria, diabetes, cardiomyopathy, scoliosis, and occasionally vision loss in late-stage disease. The discovery of the abnormal gene in FRDA and its product frataxin has provided insight into the pathophysiology and mechanisms of treatment. AREAS COVERED Although the neurologic phenotype of FRDA is well defined, there are currently no established pharmacological treatments. Omaveloxolone, a nuclear factor erythroid 2-related factor 2 (Nrf2) activator, is currently under review by the Food and Drug Administration (FDA) and has the potential to be the first approved treatment for FRDA. In the present report, we have reviewed the basic and clinical literature on Nrf2 deficiency in FRDA, and evidence for the benefit of omaveloxolone. EXPERT OPINION The present perspective suggests that omaveloxolone is a rational and efficacious therapy that is possibly disease modifying in treatment of FRDA.Palabras clave
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Triterpenos
/
Ataxia de Friedreich
Límite:
Humans
País/Región como asunto:
America do norte
Idioma:
En
Revista:
Expert Opin Investig Drugs
Asunto de la revista:
TERAPIA POR MEDICAMENTOS
Año:
2023
Tipo del documento:
Article
País de afiliación:
Estados Unidos