Long term effect of intrathecal HPBCD in patients with NP disease Type C
Based on long.term intrathecal HPBCD (IT-HPBCD) treatment of 5 patients with age of 1.4-20 years the following results were observed:
Tag: intrathecal administration
Brain uptake and distribution patterns of 2-hydroxypropyl-ß-cyclodextrin after intrathecal and intranasal administration
2-hydroxypropyl-ß-cyclodextrin (kleptose) is used for the treatment of Niemann–Pick disease. Kleptose crosses the blood–brain barrier poorly, in part because of
HPBCD can cross the Brain-Blood Barrier
Matencio et al. reported in their recent paper that 92.8% of HPBCD administered intrathecally to a child suffering from Niemann