An expanded access record providing UX003 in Mucopolysaccharidosis Type 7, sponsored by Joyce Fox. No longer available at 1 site in United States. Open to male participants. Per ClinicalTrials.gov, last updated 2017-02-06.
Sponsored by Joyce Fox · Expanded access
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.
208 studies on the registry are indexed under Rare Diseases; 112 are open to participants now.
Browse Rare Diseases studies →This is the only study on the registry with Joyce Fox as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Inclusion Criteria:
Open Label
Also known as: recombinant human beta glucuronidase, rhGUS
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No contact was published for this record. The registry link below has the sponsor’s details.
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