Transport®NPC: open phase 3 global trial of intravenous hydroxy-propyl-beta-cyclodextrin in patients with Niemann-Pick disease type C1 (NPC1)

Autor: Hastings, Caroline A., Goker-Alpan, Ozlem, Pena, Loren, Bayarri, Jordi Gascon, Martin-Hernandez, Elena, Sharma, Reena, Chacham, Orna Staretz, Spiegel, Ronen, Mengel, Eugen, Walterfang, Mark, Peters, Heidi, Fung, Victor S., Kiec-Wilk, Beata, Smith, Nicholas, Giugliani, Roberto, Guelbert, Norberto, Ezgu, Fatih, Ucar, Sema Kalkan, Murray, Bryan J., Brecht, Andreas F., Mejia, Joseph
Zdroj: In Molecular Genetics and Metabolism February 2024 141(2)
Databáze: ScienceDirect
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