Study on the Effectiveness and Safety of OTL-203 for Patients with Hurler Syndrome (MPS-IH) Compared to Standard Treatment with Stem Cell Transplantation

Registered drugNo placebo

Study on the Effectiveness and Safety of OTL-203 for Patients with Hurler Syndrome (MPS-IH) Compared to Standard Treatment with Stem Cell Transplantation

Registered drugNo placebo

Italy

Participants:0–17 years
Substances:Busulfan
Sponsor:Orchard Therapeutics (Europe) Limited

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Mucopolysaccharidosis I

Clinical trials for Mucopolysaccharidosis I

Mucopolysaccharidosis I

Study on Gene Therapy with Autologous Stem Cells and Drug Combination for Children with Mucopolysaccharidosis Type I Hurler Variant

Mucopolysaccharidosis I

Study on the Effectiveness and Safety of OTL-203 for Patients with Hurler Syndrome (MPS-IH) Compared to Standard Treatment with Stem Cell Transplantation

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