Clinical experience with miglustat therapy in pediatric patients with Niemann-Pick disease type C: A case series

  1. Pineda, M.
  2. Perez-Poyato, M.S.
  3. O'Callaghan, M.
  4. Vilaseca, M.A.
  5. Pocovi, M.
  6. Domingo, R.
  7. Ruiz Portal, L.
  8. Verdú Pérez, A.
  9. Temudo, T.
  10. Gaspar, A.
  11. Garcia Peñas, J.J.
  12. Roldán, S.
  13. Martín Fumero, L.
  14. Blanco de la Barca, O.
  15. García Silva, M.T.
  16. Macías-Vidal, J.
  17. Coll, M.J.
Journal:
Molecular Genetics and Metabolism

ISSN: 1096-7192 1096-7206

Year of publication: 2010

Volume: 99

Issue: 4

Pages: 358-366

Type: Article

DOI: 10.1016/J.YMGME.2009.11.007 GOOGLE SCHOLAR

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