Publications by authors named "Begona Arias"
Application of a simple methodology to analyze Hydroxypropyl-β-Cyclodextrin in urine using HPLC-LS in early Niemann-Pick disease type C patient.
Adrián Matencio María Agustina Alcaráz-Gómez Francisco García-Carmona Begoña Arias José Manuel López-Nicolás
J Chromatogr B Analyt Technol Biomed Life Sci
September 2018
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.
Lysosomal storage disorders (LSDs) occur at a frequency of 1 in every 5,000 live births and are a common cause of pediatric neurodegenerative disease. The relatively small number of patients with LSDs and lack of validated biomarkers are substantial challenges for clinical trial design. Here, we evaluated the use of a commercially available fluorescent probe, Lysotracker, that can be used to measure the relative acidic compartment volume of circulating B cells as a potentially universal biomarker for LSDs.
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