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NCT03604835RECRUITING

Mucopolysaccharidosis VII Disease Monitoring Program

Study Summary

The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.

Conditions
Mucopolysaccharidosis VIIMPS VIIMPS 7Sly Syndrome
Interventions / Drugs
No Intervention
View on ClinicalTrials.gov ↗
Sponsor
RARE
Ultragenyx Pharmaceutical
Rare Disease
D49/100
Study Details
Enrollment50 participants
Primary Completion—
Lead SponsorUltragenyx Pharmaceutical Inc
Data Retrieved2026-04-11 04:25:35.045716+00:00
What This Trial Means for Biotech Investors
Trial Phase Context
Status & Expected Data

Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.

Enrolled: 50 participants
Sponsor Catalyst Profile

Ultragenyx Pharmaceutical (RARE) is the sponsoring company for this trial. BiotechSign currently grades this company D (49/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

RARE Full Pipeline →All Companies →
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