Non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy (OTL-203) for Hurler syndrome.
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Tucci F, et al. Among authors: cossutta m.
Mol Ther. 2025 Sep 27:S1525-0016(25)00814-7. doi: 10.1016/j.ymthe.2025.09.042. Online ahead of print.
Mol Ther. 2025.
PMID: 41017152