Mucopolysaccharidosis Type 7
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CLINICAL_TRIALS trial_result · 2018-01-05Clinical Trial Update: 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]
Open-label Phase 1/2 trial assessing safety, efficacy, and dosing of UX003 rhGUS enzyme replacement in MPS VII has completed with results po…
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CLINICAL_TRIALS research · 2017-02-06Clinical Trial Update: An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
An open-label treatment protocol update for an advanced-stage patient with Mucopolysaccharidosis Type 7 treated with UX003 rhGUS enzyme repl…