Mucopolysaccharidosis VII Clinical Trial
Individual patient expanded access requests may be considered for patients who have no other treatment options
Expanded access may be available outside of the US in countries prior to approval by the local regulatory agencies. For full details, please visit the links provided below. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
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| Completed |
NCT02432144 -
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
|
Phase 3 | |
| Recruiting |
NCT05619900 -
Registry of Patients Diagnosed With Lysosomal Storage Diseases
|
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| Enrolling by invitation |
NCT05368038 -
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
|
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| Completed |
NCT02418455 -
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
|
Phase 2 | |
| Recruiting |
NCT03604835 -
Mucopolysaccharidosis VII Disease Monitoring Program
|
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| Recruiting |
NCT06036693 -
MPS (RaDiCo Cohort) (RaDiCo-MPS)
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