Mucopolysaccharidosis Clinical Trial
Official title:
A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7
The primary objective of the study is to evaluate the long-term safety of UX003 in subjects with MPS 7.
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. ;
| Status | Clinical Trial | Phase | |
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