CACNA1A退N--:

K Martakis1, M Giorgi2, M Spanou2

  • 1Department of Pediatric Neurology, University Children's Hospital and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; University of Cologne, Medical Faculty and University Hospital, Department of Pediatrics, Cologne, Germany.

概括

N-乙-氨酸 (NAL) 显示出治疗罕见的CACNA1A相关疾病的前景,迅速和可持续地改善着动力不良症状. 这种潜在的治疗方法在一小群患者中耐受性很好,因此需要进一步调查.