Trappsol®Cyclo™ and NPC: Efficacy shown across individual 5D domains and utilization of future assessment tools to demonstrate clinically relevant outcomes

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Bibliographic Details
Published inMolecular genetics and metabolism Vol. 144; no. 2; p. 108757
Main Authors Hastings, Caroline A., Ezgu, Fatih S., Giugliani, Roberto, Kiec-Wilk, Beata, Pawlinski, Lukasz, Mengel, Eugen, Mohand, Patricia Pérez, Ucar, Seema, Sharma, Reena, Smith, Nicholas J.C., Chien, Yin-Hsiu, Walterfang, Mark, Alsayed, Moeen, Goker-Alpan, Ozlem, Mendonca, Leonardo O., Raiman, Julian A., Spiegel, Ronen, Barone, Rita Maria Elisa, Burlina, Alberto, Calandra, Cristian R., Bayarri, Jordi Gascon, Peters, Heidi, Murray, Bryan J., Brecht, Andreas F., Mejia, Joseph
Format Journal Article
LanguageEnglish
Published Elsevier Inc 01.02.2025
Online AccessGet full text
ISSN1096-7192
DOI10.1016/j.ymgme.2024.108757

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ISSN:1096-7192
DOI:10.1016/j.ymgme.2024.108757