Overview
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Status:
No longer available
No longer available
Trial end date:
1969-12-31
1969-12-31
Target enrollment:
0
0
Participant gender:
Male
Male
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.Details
Lead Sponsor:
Joyce FoxCollaborator:
Ultragenyx Pharmaceutical Inc
Criteria
Inclusion Criteria:- 1 specific patient
- Confirmed diagnosis of MPS 7 based on leukocyte or fibroblast glucuronidase enzyme
assay or genetic testing confirming diagnosis.
- Written informed consent of parent(s) or legal guardian(s)