Sly Syndrome
2
Pipeline Programs
1
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
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1
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
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Ultragenyx PharmaceuticalUX003
Ultragenyx PharmaceuticalUX003
Clinical Trials (2)
Total enrollment: 20 patients across 2 trials
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
Start: Nov 2015Est. completion: Jan 201912 patients
Phase 3Completed
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
Start: Jul 2015Est. completion: Mar 20198 patients
Phase 2Completed
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 late-stage (Phase 3) programs — potential near-term approvals
1 companies competing in this space