Serum levels of IGF1 are a useful predictor of retinopathy of prematurity von Pérez-Muñuzuri, A, Fernández-Lorenzo, JR, Couce-Pico, ML, Blanco-Teijeiro, MJ, Fraga-Bermúdez, JM

Volltext

4CPS-031 Efficacy and safety of high-dose twice-weekly sebelipase alfa in severe-onset Wolman disease: a case report von Castro Balado, A, de Castro López, MJ, Sánchez Pintos, P, Bandín Vilar, EJ, Giráldez Montero, JM, García Quintanilla, L, Couce Pico, ML, Zarra Ferro, I

Volltext

P.40 Nutritional changes after an AAV8-mediated liver-directed gene therapy in adults with glycogen storage disease type Ia (GSD Ia) von Saavedra, H, Mount, M, Lipinski, S, Camba-Garea, M-J, de Boer, F, Boney, A, Rodriguez-Buritica, DF, Riba-Wolman, R, Ahmad, A, Pico, ML Couce, Derks, TG, Mitchell, JJ, Mitragotri, D, Grimm, AA

Volltext

P.24 - Nutritional management in adults with glycogen storage disease type Ia (GSDIa) enrolled in a phase 1/2 clinical trial of an AAV8-mediated liver-directed gene therapy von Saavedra, H, Mount, M, Lipinski, S, Camba-Garea, M-J, de Boer, F, Reineking, B, Rodriguez-Buritica, DF, Riba-Wolman, R, Ahmad, A, Pico, ML Couce, Derks, TG, Mitchell, J, Weinstein, DA, Lee, C, Valayannopoulos, V, Crombez, E

Volltext

Nutritional management in adults with glycogen storage disease type Ia (GSDIa) enrolled in a phase 1/2 clinical trial of an AAV8-mediated liver-directed gene therapy von Saavedra, H, Mount, M, Lipinski, S, Camba-Garea, M-J, de Boer, F, Reineking, B, Rodriguez-Buritica, DF, Riba-Wolman, R, Ahmad, A, Pico, ML Couce, Derks, TG, Mitchell, J, Weinstein, DA, Lee, C, Valayannopoulos, V, Crombez, E

Volltext