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

• Published in: Molecular 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
• Language: English
• Published: Elsevier Inc 01.02.2024