METHODS AND COMPOSITIONS FOR THE DIAGNOSIS AND FOR THE TREATMENT OF ADRENOLEUKODYSTROPHY
申请人:Fundació Institut d'Investigació Biomèdica
de Bellvitge (IDIBELL)
公开号:EP3118621A1
公开(公告)日:2017-01-18
The present invention is directed to a diagnostic method for adrenoleukodystrophy in a subject based on the determination of the levels of different markers. The invention also provides a method for monitoring the progression of an adrenoleukodystrophy, a method for monitoring the effect of an adrenoleukodystrophy therapy and fingolimod, an analogue, metabolite or derivative thereof, or a pharmaceutically acceptable salt thereof, for use in the treatment and/or prevention of an adrenoleukodystrophy.
本发明涉及一种基于不同标记物水平测定的受试者肾上腺脑白质营养不良症的诊断方法。本发明还提供了一种监测肾上腺白质营养不良症进展的方法、一种监测肾上腺白质营养不良症治疗效果的方法以及用于治疗和/或预防肾上腺白质营养不良症的芬戈莫德、其类似物、代谢物或衍生物或其药学上可接受的盐。