Efficacy and safety clinical trial with efavirenz in patients diagnosed with adult Niemann-pick type C with cognitive impairment

Niemann-Pick disease Type C (NPC) is a genetic, incurable, neurodegenerative disorder. This orphan disease is most frequently caused by mutations in the NPC1 protein, resulting in intralysossomal cholesterol accumulation. NPC1 is found in neuronal cell bodies, axon terminals and synaptosomes, sugges...

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Detalles Bibliográficos
Autores principales: Gascón-Bayarri, Jordi, Simon, Petru Cristian, Llop, Roser, Carnaval, Thiago, Ledesma, María Dolores, Rico, Imma, Sánchez-Castañeda, Cristina, Campdelacreu-Fumadó, Jaume, Calvo-Malvar, Nahum, Cos, Mònica, de Lama, Eugenia, Cortés-Romera, Montserrat, Rodríguez-Bel, Laura, Pérez-Sousa, Celia, Cerdán Sánchez, María, Muelas, Nuria, Sevillano, María Dolores, Mir, Pablo, López de Munain, Adolfo, Ferrer, Anna, Videla, Sebastián
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2022
Materias:
5300
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9726274/
https://www.ncbi.nlm.nih.gov/pubmed/36482560
http://dx.doi.org/10.1097/MD.0000000000031471

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