Mucopolysaccharidosis Type Hurler Syndrome
📧 Sign up to get email alerts when something new happens for Mucopolysaccharidosis Type Hurler Syndrome.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS research · 2025-12-22Clinical Trial Update: A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT [Phase 3]
This update covers a Phase 3 clinical trial evaluating OTL-203 for MPS-IH (Hurler syndrome) compared with standard of care using allogeneic …
-
CLINICAL_TRIALS research · 2025-12-09Clinical Trial Update: Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant [Phase 1, Phase 2]
Clinical trial update on a gene therapy using modified autologous hematopoietic stem cells to treat Mucopolysaccharidosis type I, Hurler syn…