赫勒综合症 葡萄糖氨基甘油 脑脊液的减少 没有针对大脑的治疗

Troy C Lund1, Elizabeth Braunlin2, Lynda E Polgreen3

  • 1Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota, Minneapolis, Minnesota, USA.

Annals of neurology
|September 7, 2023
PubMed
概括

新的赫勒综合征疗法旨在降解大脑中的GAG. 然而,在接受标准酶替代疗法的患者中,脑脊液GAG的降低对其作为血脑屏障透标志物的使用提出了质疑.