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

Joyce Fox

Status

Conditions: Mucopolysaccharidosis Type 7

Treatments: Drug: UX003

Study type: Expanded Access

Funder types: Other; Industry

Identifiers

NCT02097251

13-606A

Details and patient eligibility

About

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.

Sex: Male

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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)