Mucopolysaccharidosis Type Vi
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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 · 2026-02-11Clinical 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 · 2026-02-02Clinical 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 · 2015-08-04Clinical Trial Update: Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
A completed clinical trial evaluating diagnostic approaches for mucopolysaccharidosis type IV A and type VI in patients presenting with bila…