Clinical Trial: An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Study Status: No longer available
Recruit Status: No longer available
Study Type: Expanded Access
Official Title: An Open-Label Treatment Protocol With UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy for an Advanced Stage Mucopolysaccharidosis Type 7 (
Brief 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.
Detailed Summary:
Sponsor: Joyce Fox
Current Primary Outcome:
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Northwell Health
Dates:
Date Received: March 24, 2014
Date Started:
Date Completion:
Last Updated: February 2, 2017
Last Verified: February 2017