Unité des Ataxies Cérébelleuses, Department of Neurology, Médiathèque Jean Jacquy, CHU-Charleroi, 6000 Charleroi, Belgium.
Department of Neurosciences, Université de Mons, 7034 Mons, Belgium.
Int J Environ Res Public Health. 2021 Sep 24;18(19):10057. doi: 10.3390/ijerph181910057.
Cerebellar ataxias (CAs) manifest with a combination of motor incoordination, cognitive, affective and recently identified social symptoms. Novel therapies aim to stop the progression of the subgroup of the degenerative ataxias, or even to cure the disease with a functional and anatomical restoration of the cerebellar circuitry in the near future. The goal of stopping the progression of the disease is particularly relevant if applied at a very early stage of the disease, when the cerebellar reserve is only slightly impaired. Therefore, the search of the prodromal phase or pre-ataxic stage of CAs represents a very important challenge for the scientific community. The identification of pre-manifest individuals and the recruitment of individuals at risk has become a key-challenge to address neuroprotective therapies. The feasibility is high due to the recent progress in the biological and morphological biomarkers of CAs.
小脑共济失调（CAs）表现为运动协调障碍、认知、情感和最近发现的社交症状的组合。新型疗法旨在阻止退行性共济失调亚组的进展，甚至在不久的将来通过恢复小脑回路的功能和解剖结构来治愈疾病。如果在疾病的早期阶段应用，当小脑储备功能只是轻微受损时，阻止疾病进展的目标尤为重要。因此，寻找 CAs 的前驱期或前共济失调期是科学界面临的一个非常重要的挑战。识别出无症状个体并招募处于风险中的个体已成为解决神经保护疗法的关键挑战。由于最近在 CAs 的生物学和形态学生物标志物方面取得了进展，因此该方法具有很高的可行性。
