Browsing by Author "Gascón Bayarri, Jordi"

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

      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 Arregui, Adolfo José; Ferrer, Anna; Videla, Sebastián (Wolters Kluwer Health, 2022-12)
      Background: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 ...