Twenty-five patients with Niemann Pick disease type C (age range: 7 months to 44 years) were enrolled in an Italian independent multicenter trial and treated with miglustat for periods from 48 to 96 months.

Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C / Fecarotta, S., Romano, A., DELLA CASA, R., DEL GIUDICE, E., Bruschini, D., Mansi, G., Bembi, B., Dardis, A., Fiumara, A., Di Rocco, M., Uziel, G., Ardissone, A., Roccatello, D., Alpa, M., Bertini, E., D'Amico, A., Dionisi Vici, C., Deodato, F., Caviglia, S., Federico, A., et al.. - In: ORPHANET JOURNAL OF RARE DISEASES. - ISSN 1750-1172. - 10:1(2015), pp. 22-32. [10.1186/s13023-015-0240-y]

Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C

FECAROTTA, SIMONA;ROMANO, ALFONSO;DELLA CASA, ROBERTO;DEL GIUDICE, ENNIO;MANSI, GIUSEPPINA;DEODATO, FEDERICA;FEDERICO, ANTONIO;FILLA, ALESSANDRO;Russo, Cinzia;PARENTI, GIANCARLO;ANDRIA, GENEROSO
2015

Abstract

Twenty-five patients with Niemann Pick disease type C (age range: 7 months to 44 years) were enrolled in an Italian independent multicenter trial and treated with miglustat for periods from 48 to 96 months.
2015
Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C / Fecarotta, S., Romano, A., DELLA CASA, R., DEL GIUDICE, E., Bruschini, D., Mansi, G., Bembi, B., Dardis, A., Fiumara, A., Di Rocco, M., Uziel, G., Ardissone, A., Roccatello, D., Alpa, M., Bertini, E., D'Amico, A., Dionisi Vici, C., Deodato, F., Caviglia, S., Federico, A., et al.. - In: ORPHANET JOURNAL OF RARE DISEASES. - ISSN 1750-1172. - 10:1(2015), pp. 22-32. [10.1186/s13023-015-0240-y]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11588/621854
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