AI Article Synopsis
- A new high-resolution liquid chromatography method was used to analyze Hydroxypropyl-beta-Cyclodextrin (HPβCD) in a child with Niemann-Pick Type C disease.
- The treatment not only halted the disease's progression but improved the patient's life expectancy and quality of life.
- The pharmacokinetics of HPβCD indicated a 92.8% recovery rate, with no detectable levels in urine after 88 hours, and treatments can now be spaced every four days instead of the previous two-week interval without showing toxicity.
Article 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.
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| http://dx.doi.org/10.1016/j.jchromb.2018.06.051 | DOI Listing |
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