Mechanistic convergence and shared therapeutic targets in Niemann-Pick disease.
Journal Of Inherited Metabolic Disease
Colaco, Alexandria A; Kaya, Ecem E; Adriaenssens, Elias E; Davis, Lianne C LC; Zampieri, Stefania S; Fernández-Suárez, María E ME; Tan, Chong Y CY; Deegan, Patrick B PB; Porter, Forbes D FD; Galione, Antony A; Bembi, Bruno B; Dardis, Andrea A; Platt, Frances M FM