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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

Paul J. Orchard,Robert F. Wynn,Sandhya Kharbanda,Rebecca Ahrens-Nicklas,Peter M. van Hasselt,Troy C. Lund,Timothy S. Olson,Francesca Tucci, Leonie Martin, Nathalie Boeglin,Jean Brooks, Su Syonmez,Laura Campbell,Paul Harmatz,Simon A. Jones,Ashish O. Gupta,Maria Ester Bernardo

Molecular Genetics and Metabolism（2024）

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关键词
Hematopoietic Cell Transplantation
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