Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice
2020 · Science Translational Medicine · 197 citations
https://doi.org/10.1126/scitranslmed.aay1163
J
Julie C. Ullman
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Julie C. Ullman is an academic researcher from Denali Therapeutics (United States). The author has contributed to research in topics: Lysosomal Storage Disorders Research & Glycogen Storage Diseases and Myoclonus & Cellular transport and secretion. The author has an h-index of 11, co-authored 28 publications.
ORCID: 0000-0003-1325-0650Papers by this author
worksHigh-Throughput Liquid Chromatography–Tandem Mass Spectrometry Quantification of Glycosaminoglycans as Biomarkers of Mucopolysaccharidosis II
2020 · International Journal of Molecular Sciences · 18 citations
https://doi.org/10.3390/ijms21155449
Small molecule inhibition of glycogen synthase I reduces muscle glycogen content and improves biomarkers in a mouse model of Pompe disease
2024 · American Journal of Physiology-Endocrinology and Metabolism · 5 citations
https://doi.org/10.1152/ajpendo.00175.2024
First‐in‐Human Evaluation of Safety, Pharmacokinetics and Muscle Glycogen Lowering of a Novel Glycogen Synthase 1 Inhibitor for the Treatment of Pompe Disease
2024 · Clinical Pharmacology & Therapeutics · 5 citations
https://doi.org/10.1002/cpt.3470
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