Allogeneic hematopoietic stem cell transplantation modulates neurodevelopmental trajectories in mucopolysaccharidosis: a longitudinal study of subtype-specific outcomes and age-dependent efficacy
HSCT stabilizes/improves neurodevelopment in MPS, modulated by subtype and transplant age. Early intervention optimizes outcomes, particularly for MPS III. Culturally adapted GDS-C enables precise monitoring, guiding HSCT timing and rehabilitation.
Authors
- Yang Xu (ORCID: https://orcid.org/0000-0002-0958-8547)
- Yunhui Mi
- Jianmin Wang (ORCID: https://orcid.org/0000-0002-5994-3359)
- Minxue Shen (ORCID: https://orcid.org/0000-0003-0441-9303)
- Jing Xie (ORCID: https://orcid.org/0009-0003-2142-4531)
- Xi Fang (ORCID: https://orcid.org/0000-0002-6692-5730)
- Chen Zhou (ORCID: https://orcid.org/0009-0008-7304-7439)
- Chengjuan Luo
Institutions
- Shanghai Jiao Tong University (CN)
- XinHua Hospital (CN)
- Shanghai Children's Medical Center (CN)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-11-19
- DOI
- https://doi.org/10.1186/s13023-025-04025-3
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00