Hurler Syndrome - 22 Studies Found
| Recruiting |
: Mucopolysaccharidosis I (MPS I) Registry : Mucopolysaccharidosis I (MPS I) : 2005-09-02 |
| RECRUITING |
: A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT : MPS-IH (Hurler Syndrome) : 2024-11-21 : Experimental: OTL-203: Autologous CD34+ enriched cell fraction that contains hematopoietic stem and progenitor cells transduced ex vivo using lentivir |