Not logged inIntrathecal idursulfase-IT in children younger than 3 years with neuronopathic mucopolysaccharidosis II in a single-arm, open-label, phase 2/3 substudy and extension
Muenzer, Joseph; Burton, Barbara K.; Harmatz, Paul; Gutierrez-solana, Luis Gonzalez; Ruiz-garcia, Matilde; Jones, Simon A.; Guffon, Nathalie; Inbar-feigenberg, Michal; Bratkovic, Drago; Rust, Stewart; Hale, Michael; Wu, Yuna; Yee, Karen S.; Whiteman, David A. H.; Alexanderian, David .png)
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SaveClinical, biochemical, and molecular studies in pyridoxine-dependent epilepsy. Antisense therapy as possible new therapeutic optionPerez, Belen; Gonzalez Gutierrez-Solana, Luis; Verdu, Alfonso; Merinero, Begona; Yuste-Checa, Patricia; Ruiz-Sala, Pedro; Calvo, Rocio; Jalan, Anil; Lopez Marin, Laura; Campos, Oscar; Angeles Ruiz, Maria; San Miguel, Marta; Vazquez, Maria; Castro, Margarita; Ferrer, Isaac; Navarrete, Rosa; Ruiz Desviat, Lourdes; Lapunzina, Pablo; Ugarte, Magdalena; Perez-Cerda, Celia Share Save Glial fibrillary acidic protein mutations in infantile, juvenile, and adult forms of Alexander diseaseLi, R; Johnson, AB; Salomons, G; Goldman, JE; Naidu, S; Quinlan, R; Cree, B; Ruyle, SZ; Banwell, B; D'Hooghe, M; Siebert, JR; Rolf, CM; Cox, H; Reddy, A; Gutiérrez-Solana, LG; Collins, A; Weller, RO; Messing, A; van der Knaap, MS; Brenner, M Share Save 
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