1.
J Chromatogr B Analyt Technol Biomed Life Sci
; 1093-1094: 47-51, 2018 Sep 01.
Article
in English
| MEDLINE
| ID: mdl-29990712
ABSTRACT
A new methodology, based on high resolution liquid chromatography with light scatterin detector, is applied to analyze Hydroxypropyl-beta-Cyclodextrin (HPßCD) in urine samples of a child affected by Niemann-Pick Type C disease. The treatment not only stopped disease progression, but has also increased the life expectancy and quality of our patient. The pharmacokinetic of HPßCD in the patient was studied with a 92.8% of HPßCD recovered. At 88â¯h, no HPßCD was found in the urine. During the treatment, HPßCD has not shown toxicity. Before application of the new treatment, injections were given every two weeks but, we have demonstrated that this can be increased to every four days.