Application of N-palmitoyl-O-phosphocholineserine for diagnosis and assessment of response to treatment in Niemann-Pick type C disease
Rohini Sidhu, Pamela Kell, Dennis J. Dietzen, Nicole Y. Farhat, An Dang, Forbes D. Porter, Elizabeth Berry‐Kravis, Charles H. Vite, Janine Reunert, Thorsten Marquardt, Roberto Giugliani, Charles Marques Lourenço, Olaf A. Bodamer, Raymond Wang, Ellen Plummer, Jean E. Schaffer, Daniel Ory, Xuntian Jiang
Topics & Concepts
NPC1Niemann–Pick disease, type CInternal medicineCerebrospinal fluidGastroenterologyMedicineDiseaseBiomarkerFabry diseaseEndocrinologyChemistryBiochemistryEndosomeReceptorLysosomal Storage Disorders ResearchCarbohydrate Chemistry and SynthesisAutoimmune and Inflammatory Disorders Research