公开(公告)号：CA3062255A1

公开(公告)日：2018-11-08

申请号：CA3062255

申请日：2018-05-04

Applicant: STICHTING VUMC

Inventor： VAN DER KNAAP MARGO SIENTJE ,  HEINE VIVI MAJELLA ,  ABBINK GERTRUDE ELISE MARIA

IPC: A61K31/00 ,  A61K31/4168 ,  A61P25/28

Abstract: The invention relates to Guanabenz or an analogue thereof for use in the treatment and to methods of treatment of a subject that has vanishing white matter (VWM), comprising administering the compound Guanabenz or an analogue thereof to the subject in need thereof. Also provided are methods with which the success of a medical intervention in a subject with VWM can be determined. Such methods comprise quantifying the translocation of Bergmann glia into the molecular layer in the cerebellum in a suitable sample of the sub ject. The sample is typically a post mortem sample.