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 (MPS 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