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Hurler Syndrome - 22 Studies Found
| Estado | Estudiar |
| Completed |
Nombre del estudio: Mucopolysaccharidosis I (MPS I) Registry Condición: Mucopolysaccharidosis I (MPS I) Fecha: 2005-09-02 |
| Completed |
Nombre del estudio: A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT Condición: MPS-IH (Hurler Syndrome) Fecha: 2025-11-04 Intervenciones: Experimental: OTL-203: Autologous CD34+ enriched cell fraction that contains hematopoietic stem and progenitor cells transduced ex vivo using lentivir |