Mucopolysaccharidosis Type 7
📧 Sign up to get email alerts when something new happens for Mucopolysaccharidosis Type 7.
Sign up freeTop drugs studied for this disease
-
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…