Mucopolysaccharidosis Type VII (DBCOND0063470)
Identifiers
- Synonyms
- MPS VII / Mucopolysaccharidosis VII / Mucopolysaccharidosis Type 7 / Mucopolysaccharidosis type VII / Other mucopolysaccharidoses / Mucopolysaccharidosis, MPS-VII (disorder) / Deficiency of beta-glucuronidase (disorder) / Sly's syndrome
Associated Data
- Indicated Drugs and Targets
Drug Description Targets Vestronidase alfa A recombinant beta-glucuronidase used to treat mucopolysaccharidosis VII.No drug targets - Clinical Trials
Identifier Title Drug(s) Purpose Phase Status NCT04532047 In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases treatment 1 recruiting NCT03604835 Mucopolysaccharidosis VII Disease Monitoring Program No drug interventions Not Available Not Available recruiting NCT03775174 Expanded Access to Mepsevii Not Available Not Available available NCT02418455 Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age treatment 2 completed NCT02230566 A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) treatment 3 completed NCT02432144 A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) treatment 3 completed NCT01870375 Longitudinal Studies of Brain Structure and Function in MPS Disorders No drug interventions Not Available Not Available completed NCT05368038 ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program No drug interventions Not Available Not Available enrolling_by_invitation NCT06036693 MPS (RaDiCo Cohort) (RaDiCo-MPS) No drug interventions Not Available Not Available recruiting NCT05619900 Registry of Patients Diagnosed With Lysosomal Storage Diseases No drug interventions Not Available Not Available recruiting NCT05687474 Baby Detect : Genomic Newborn Screening No drug interventions Not Available Not Available recruiting NCT03655223 Early Check: Expanded Screening in Newborns No drug interventions Not Available Not Available enrolling_by_invitation NCT02097251 An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient Not Available Not Available no_longer_available NCT01856218 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) treatment 1 / 2 completed