An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
NCT02097251 · Status: NO_LONGER_AVAILABLE · Type: EXPANDED_ACCESS
Last updated 2017-02-06
Summary
Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up to 144 weeks.
Conditions
- Mucopolysaccharidosis Type 7
Interventions
- DRUG
-
UX003
Open Label
Sponsors & Collaborators
- collaborator INDUSTRY
-
Joyce Fox
lead OTHER
Eligibility
- Sex
- MALE
- Healthy Volunteers
- No
Timeline & Regulatory
Countries
- United States
Study Locations
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