Gascon-Bayarri, Jordi; Rico, Inmaculada; Sanchez-Castaneda, Cristina; Ledesma, Maria Dolores; Carnaval, Thiago; Bejr-Kasem, Helena; Campdelacreu, Jaume; Ferrer, Anna; Rodriguez-Bel, Laura; Cos, Monica; de Lama, Eugenia; de Munain, Adolfo Lopez; Rouco, Idoia; Perez-Sousa, Celia; Cerdan, Maria; Muelas, Nuria; Sevillano, Maria Dolores; Mir, Pablo; Villoria, Jesus; Videla, Sebastian
(ELSEVIER SCIENCE INC, 2025-09)
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 ...