Tada Masayoshi, Yokoseki Akio, Onodera Osamu
Department of Neurology, Brain Research Institute, Niigata University.
Brain Nerve. 2017 Aug;69(8):879-890. doi: 10.11477/mf.1416200839.
Hereditary spinocerebellar degenerations (SCD) are a group of neurodegenerative disorders characterized by slowly progressive ataxia associated with non-cerebellar neurological signs and symptoms. In the Japanese population, dominantly inherited SCDs are much more common than recessively inherited or X-linked SCDs. The most common dominantly inherited SCD in Japan, as well as in many other countries, is Machado-Joseph disease, also known as spinocerebellar ataxia type 3 (MJD/SCA3). MJD/SCA3 is frequently accompanied by non-cerebellar symptoms, including progressive external ophthalmoplegia, pyramidal signs, dystonia, rigidity, dysarthria, and distal muscle atrophies. SCA6 and SCA31 represent a pure cerebellar subtype of SCD, occasionally accompanied by non-cerebellar signs. Detailed medical history and neurological examination are important for clinicians to diagnose hereditary SCDs, although genetic testing can help confirm the diagnosis. Despite increasing understanding of the molecular mechanisms underlying these fatal diseases, preventive therapies are currently lacking.
遗传性脊髓小脑变性(SCD)是一组神经退行性疾病,其特征为缓慢进展的共济失调,并伴有非小脑性神经体征和症状。在日本人群中,显性遗传性SCD比隐性遗传性或X连锁SCD更为常见。在日本以及许多其他国家,最常见的显性遗传性SCD是马查多-约瑟夫病,也称为脊髓小脑共济失调3型(MJD/SCA3)。MJD/SCA3常伴有非小脑症状,包括进行性眼外肌麻痹、锥体束征、肌张力障碍、僵硬、构音障碍和远端肌肉萎缩。SCA6和SCA31代表SCD的纯小脑亚型,偶尔伴有非小脑体征。详细的病史和神经系统检查对临床医生诊断遗传性SCD很重要,尽管基因检测有助于确诊。尽管对这些致命疾病的分子机制的了解不断增加,但目前仍缺乏预防性治疗方法。