Mucopolysaccharidosis Type 7
Pipeline by Development Stage
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (2)
Total enrollment: 3 patients across 2 trials
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)
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Phase Legend
Key Insights
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