Mucopolysaccharidosis Type
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-03-30Clinical Trial Update: ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I [Phase 1]
Phase 1 clinical trial update for ISP-001, Immusoft's Sleeping Beauty transposon-engineered B cells intended to treat Mucopolysaccharidosis …
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) [Phase 1]
Phase 1 prenatal enzyme replacement therapy trial PEARL is currently recruiting for multiple lysosomal storage disorders including MPS I, MP…
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CLINICAL_TRIALS research · 2026-03-12Clinical Trial Update: RGX-111 Gene Therapy in Patients With MPS I [Phase 1, Phase 2]
The RGX-111 gene therapy trial for Mucopolysaccharidosis Type I is currently suspended in Phase 1/2.
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: MPS (RaDiCo Cohort) (RaDiCo-MPS)
An update on the RaDiCo-MPS clinical trial cohort sponsored by INSERM, recruiting patients with several mucopolysaccharidosis types I-IX and…
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CLINICAL_TRIALS research · 2026-02-09Clinical Trial Update: Longitudinal Study of Neurodegenerative Disorders
This clinical trial update describes a longitudinal study of several neurodegenerative and lysosomal storage disorders. The study is current…
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CLINICAL_TRIALS research · 2026-02-02Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI [Phase 1, Phase 2]
A clinical trial is evaluating the safety and efficacy of adalimumab in mucopolysaccharidosis types I, II, and VI; the study is currently ac…
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CLINICAL_TRIALS research · 2026-01-26Clinical Trial Update: Cardiac Structure and Function in MPS
This clinical trial update shows ongoing recruitment for a study evaluating cardiac structure and function in patients with mucopolysacchari…
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CLINICAL_TRIALS research · 2025-10-30Clinical Trial Update: Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
This clinical trial update evaluates intravenous laronidase pharmacokinetics before and after hematopoietic cell transplantation in patients…