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About
The primary objective of the study is to evaluate the long-term safety of UX003 in subjects with MPS 7.
Full description
Participants with MPS 7 who were UX003 treatment-naïve or previously enrolled and treated in a prior clinical study of UX003 (e.g. UX003-CL301 [NCT02230566], investigator sponsored trials, expanded access/compassionate use) can enroll into this treatment and extension study provided all eligibility criteria is met for a given participant.
Enrollment
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Primary purpose
Allocation
Interventional model
Masking
12 participants in 1 patient group
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Data sourced from clinicaltrials.gov
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