Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C
- 期刊名称Orphanet Journal of Rare Diseases
- 作者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
- 卷第10卷
- 期第1期
- 印刷ISSN1750-1172
- 文件大小1424.77KB
- 发表时间2015
- DOI10.1186/s13023-015-0240-y
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