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Results of a Global Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Sebelipase Alfa as an Enzyme Replacement Therapy in Children and Adults with Lysosomal Acid Lipase Deficiency Balwani, M., Burton, B., Baric, I., Bialer, M., Burrow, T. A., Camarena Grande, C., Coker, M., Consuelo Sanchez, A., Deegan, P., Di Rocco, M., Enns, G. M., Erbe, R. W., Ezgu, F., Feillet, F., Ficicioglu, C., Furuya, K. N., Bernardo Guelbert, N., Kane, J., Kostyleva, M., Laukaitis, C. M., Malinova, V., Mengel, E., Murphy, E., Neilan, E. G., Nightingale, S., Pastor Rosado, J., Peters, H., Rahman, Y., Scarpa, M., Schwab, K., Smolka, V., Taybert, J., Valayannopoulos, V., Wood, M., Zeniya, M., Yang, Y., Eckert, S., Rojas-Caro, S., Quinn, A. G. WILEY-BLACKWELL. 2014: 1270A
View details for Web of Science ID 000345517000006