NO_LONGER_AVAILABLENot Applicable

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

United States

Plain-language 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.

Who can participate

SexMALE

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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)

Trial details

NCT IDNCT02097251

SponsorJoyce Fox

Sponsor typeOTHER

Study typeEXPANDED_ACCESS

View on ClinicalTrials.gov More Mucopolysaccharidosis Type 7 trials