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Repurposing Dipyridamole in Niemann Pick Type C Disease: A Proof of Concept Study

Niemann Pick type C disease (NPC) is a rare disorder characterized by lysosomal lipid accumulation that damages peripheral organs and the central nervous system. Currently, only miglustat is authorized for NPC treatment in Europe, and thus the identification of new therapies is necessary. The hypoth...

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Detalles Bibliográficos
Autores principales: Pepponi, Rita, De Simone, Roberta, De Nuccio, Chiara, Visentin, Sergio, Matteucci, Andrea, Bernardo, Antonietta, Popoli, Patrizia, Ferrante, Antonella
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8999038/
https://www.ncbi.nlm.nih.gov/pubmed/35408815
http://dx.doi.org/10.3390/ijms23073456