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Mucopolysaccharidosis VII Disease Monitoring Program
Sponsor: Ultragenyx Pharmaceutical Inc
Summary
The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.
Official title: Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
50
Start Date
2018-01-29
Completion Date
2033-05
Last Updated
2026-03-17
Healthy Volunteers
No
Interventions
No Intervention
Access to any treatment is through authorized commercial use or available expanded access programs only and not as a part of this DMP.
Locations (14)
Children's Hospital of Orange County
Orange, California, United States
Children's National Health System
Washington D.C., District of Columbia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Michigan
Ann Arbor, Michigan, United States
New York University Langone Medical Center
New York, New York, United States
University of Utah Medical Center
Salt Lake City, Utah, United States
Seattle Children's Hospital
Seattle, Washington, United States
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
Buenos Aires, Argentina
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil
Centre Hospitalier Universitaire La Timone
Marseille, Provence-Alpes-Côte d'Azur Region, France
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Mainz, Germany
Erasmus University Medical Center Rotterdam
Rotterdam, South Holland, Netherlands
Centro Hospitalar do Porto
Porto, Portugal
Hospital Universitario Virgen del Rocío Pabellón Infantil
Seville, Spain