P037: Design of a multi-center randomized phase 3 clinical trial (HURCULES) evaluating OTL-203 in MPS-IH vs allogeneic hematopoietic stem cell transplantation

oleh: Paul Harmatz, Robert Wynn, Ashish Gupta, Sandhya Kharbanda, Caroline Lindemans, Rebecca Ahrens-Nicklas, Peter van Hasselt, Troy Lund, Timothy Olson, Francesca Tucci, Leonie Martin, Nathalie Boeglin, Jean Brooks, Su Syonmez, Laura Campbell, Simon Jones, Paul Orchard, Maria Ester Bernardo