Clinical experience with miglustat therapy in pediatric patients with Niemann–Pick disease type C: A case series
| Autor: | Pineda, M., Perez-Poyato, M.S., O’Callaghan, M., Vilaseca, M.A., Pocovi, M., Domingo, R., Ruiz Portal, L., Verdú Pérez, A., Temudo, T., Gaspar, A., Garcia Peñas, J.J., Roldán, S., Martín Fumero, L., Blanco de la Barca, O., García Silva, M.T., Macías-Vidal, J., Coll, M.J. |
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| Zdroj: | In Molecular Genetics and Metabolism 2010 99(4):358-366 |
| Databáze: | ScienceDirect |
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