Mucopolysaccharidosis Type Iv
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CLINICAL_TRIALS other · 2026-04-08Clinical Trial Update: Registry of Patients Diagnosed With Lysosomal Storage Diseases
This ClinicalTrials.gov registry entry describes ongoing recruitment for a registry of patients diagnosed with various lysosomal storage dis…
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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 · 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…
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CLINICAL_TRIALS research · 2013-12-04Clinical Trial Update: Psychological Concomitants of Morquio Syndrome (The MAP Study)
A clinical trial update describes the MAP Study on psychological concomitants in Morquio syndrome (MPS IV), which has completed.
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