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Efficacy and safety of efavirenz in Niemann-Pick disease type C

Introduction: In search of disease-modifying treatments for the Niemann-Pick disease type C (NPC), this Phase II single-arm clinical trial evaluated the safety and efficacy of efavirenz, a reverse transcriptase inhibitor that potentially ameliorates neuronal cholesterol turnover, typically impaired...

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Autori principali: Jordi Gascón-Bayarri, Inmaculada Rico, Cristina Sánchez-Castañeda, María Dolores Ledesma, Thiago Carnaval, Helena Bejr-Kasem, Jaume Campdelacreu, Anna Ferrer, Laura Rodríguez-Bel, Mónica Cos, Eugenia de Lama, Adolfo López de Munain, Idoia Rouco, Celia Pérez-Sousa, María Cerdán, Nuria Muelas, María Dolores Sevillano, Pablo Mir, Jesús Villoria, Sebastián Videla
Natura: Artigo
Lingua:Inglês
Pubblicazione: Elsevier 2025-09-01
Serie:Neurotherapeutics
Soggetti:
Clinical trial
Dementia
Lipid metabolism disorders
Lysosomal storage diseases
Neurodegenerative diseases
Neuropsychological tests
Accesso online:http://www.sciencedirect.com/science/article/pii/S1878747925001849
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