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Results for “MPS 7”

6 trials

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Showing 6 of 6 results

Testing effectiveness (Phase 2)Study completedNCT02418455
What this trial is testing

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

Who this might be right for
Sly SyndromeMPS VIIMucopolysaccharidosis+1 more
Ultragenyx Pharmaceutical Inc 8
Not applicableNo Longer AvailableNCT02097251
What this trial is testing

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

Who this might be right for
Mucopolysaccharidosis Type 7
Joyce Fox
Testing effectiveness (Phase 2)Study completedNCT01856218
What this trial is testing

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Who this might be right for
Mucopolysaccharidosis Type 7
Ultragenyx Pharmaceutical Inc 3
Large-scale testing (Phase 3)Study completedNCT02432144
What this trial is testing

UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Who this might be right for
Sly SyndromeMPS VIIMucopolysaccharidosis+1 more
Ultragenyx Pharmaceutical Inc 12
Large-scale testing (Phase 3)Study completedNCT02230566
What this trial is testing

UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Who this might be right for
MPS 7Sly SyndromeMucopolysaccharidosis+1 more
Ultragenyx Pharmaceutical Inc 12
Not applicableLooking for participantsNCT03604835
What this trial is testing

Mucopolysaccharidosis VII Disease Monitoring Program

Who this might be right for
Mucopolysaccharidosis VIIMPS VIIMPS 7+1 more
Ultragenyx Pharmaceutical Inc 50