Mucopolysaccharidosis VII

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Information
Disease name
Mucopolysaccharidosis VII
Disease ID
Description
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
Genes Mutation Description Source Links
NCT ID Status Phase Summary Start date Completion date
NCT03775174 Available Expanded Access to Mepsevii
NCT02432144 Completed Phase 3 A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) November 10, 2015 January 14, 2019
NCT02418455 Completed Phase 2 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 July 21, 2015 March 26, 2019
NCT05368038 Enrolling by invitation ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program May 10, 2021 July 31, 2026
NCT03604835 Recruiting Mucopolysaccharidosis VII Disease Monitoring Program January 29, 2018 May 2033
NCT05619900 Recruiting Registry of Patients Diagnosed With Lysosomal Storage Diseases May 31, 2022 May 31, 2050
NCT05687474 Recruiting Baby Detect : Genomic Newborn Screening September 1, 2022 August 31, 2025
NCT06036693 Recruiting MPS (RaDiCo Cohort) (RaDiCo-MPS) December 20, 2017 December 20, 2024
MeSH unique ID (MeSH (Medical Subject Headings))
D016538