Design of a multi-center randomized active controlled phase 3 clinical trial (HURCULES) evaluating the safety and efficacy of OTL-203 in patients with MPS IH versus standard of care with allogeneic hematopoietic stem cell transplantation

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Bibliographic Details
Published inMolecular genetics and metabolism Vol. 141; no. 2; p. 107988
Main Authors Orchard, Paul J., Wynn, Robert F., Kharbanda, Sandhya, Ahrens-Nicklas, Rebecca, van Hasselt, Peter M., Lund, Troy C., Olson, Timothy S., Tucci, Francesca, Martin, Leonie, Boeglin, Nathalie, Brooks, Jean, Syonmez, Su, Campbell, Laura, Harmatz, Paul, Jones, Simon A., Gupta, Ashish O., Bernardo, Maria Ester
Format Journal Article
LanguageEnglish
Published Elsevier Inc 01.02.2024
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2023.107988