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, Simona; Romano, Alfonso; DELLA CASA, Roberto; DEL GIUDICE, Ennio; Bruschini, Diana; Mansi, Giuseppina; Bembi, Bruno; Dardis, Andrea; Fiumara, Agata; Di Rocco, Maja; Uziel, Graziella; Ardissone, Anna; Roccatello, Dario; Alpa, Mirella; Bertini, Enrico; D'Amico, Adele; Dionisi Vici, Carlo; Deodato, Federica; Caviglia, Stefania; Federico, Antonio; Palmeri, Silvia; Gabrielli, Orazio; Santoro, Lucia; Filla, Alessandro; Russo, Cinzia; Parenti, Giancarlo; Andria, Generoso. - 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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
