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Clinical Trial 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.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT02097251
Study type Expanded Access
Source Northwell Health
Contact
Status No longer available
Phase N/A

See also
  Status Clinical Trial Phase
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Completed NCT01856218 - An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) Phase 1/Phase 2