Overview

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

Status:
No longer available
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
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 Fox
Collaborator:
Ultragenyx Pharmaceutical Inc