Neurológia pre prax 2/2026
Novel therapeutic options in hereditary neurodegenerative cerebellar ataxias
This review summarizes current options for both disease-modifying and symptomatic treatment of degenerative cerebellar ataxias, with a particular focus on two conditions that are significantly reshaping everyday neurological practice: Friedreich’s ataxia (FA), with the recent availability of the targeted therapy omaveloxolone, and spinocerebellar ataxia type 27B (SCA27B), for which accumulating evidence supports the efficacy of 4-aminopyridine (4-AP).
Keywords: hereditary cerebellar ataxias, Friedreich’s ataxia, spinocerebellar ataxia type 27B, SCA27B, 4-aminopyridine, omaveloxolone.