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Application of a simple methodology to analyze Hydroxypropyl-β-Cyclodextrin in urine using HPLC-LS in early Niemann-Pick disease type C patient.
Application of a simple methodology to analyze Hydroxypropyl-β-Cyclodextrin in urine using HPLC-LS in early Niemann-Pick disease type C patient.
- Source :
-
Journal of chromatography. B, Analytical technologies in the biomedical and life sciences [J Chromatogr B Analyt Technol Biomed Life Sci] 2018 Sep 01; Vol. 1093-1094, pp. 47-51. Date of Electronic Publication: 2018 Jun 25. - Publication Year :
- 2018
-
Abstract
- A new methodology, based on high resolution liquid chromatography with light scatterin detector, is applied to analyze Hydroxypropyl-beta-Cyclodextrin (HPβCD) 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 HPβCD in the patient was studied with a 92.8% of HPβCD recovered. At 88 h, no HPβCD was found in the urine. During the treatment, HPβCD 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.<br /> (Copyright © 2018 Elsevier B.V. All rights reserved.)
- Subjects :
- Child, Preschool
Humans
Light
Orphan Drug Production
Reproducibility of Results
Scattering, Radiation
2-Hydroxypropyl-beta-cyclodextrin chemistry
2-Hydroxypropyl-beta-cyclodextrin pharmacokinetics
2-Hydroxypropyl-beta-cyclodextrin therapeutic use
2-Hydroxypropyl-beta-cyclodextrin urine
Anticholesteremic Agents chemistry
Anticholesteremic Agents pharmacokinetics
Anticholesteremic Agents therapeutic use
Anticholesteremic Agents urine
Chromatography, High Pressure Liquid methods
Niemann-Pick Disease, Type C drug therapy
Niemann-Pick Disease, Type C metabolism
Subjects
Details
- Language :
- English
- ISSN :
- 1873-376X
- Volume :
- 1093-1094
- Database :
- MEDLINE
- Journal :
- Journal of chromatography. B, Analytical technologies in the biomedical and life sciences
- Publication Type :
- Academic Journal
- Accession number :
- 29990712
- Full Text :
- https://doi.org/10.1016/j.jchromb.2018.06.051