Scientists track rare disease to improve care
NCT ID NCT03604835
First seen Oct 31, 2025 · Last updated May 19, 2026 · Updated 26 times
Summary
This study follows up to 50 people with MPS VII (Sly Syndrome) to understand how the disease changes over time. Researchers will also check how well and how safely the drug vestronidase alfa works in the long run. Participants may or may not be taking this treatment. The goal is to learn more about the disease and improve future care.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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Centre Hospitalier Universitaire La Timone
RECRUITINGMarseille, Provence-Alpes-Côte d'Azur Region, 13005, France
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Centro Hospitalar do Porto
RECRUITINGPorto, 4050-651, Portugal
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Children's Hospital of Orange County
RECRUITINGOrange, California, 92868, United States
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Children's National Health System
RECRUITINGWashington D.C., District of Columbia, 20010, United States
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Erasmus University Medical Center Rotterdam
TERMINATEDRotterdam, South Holland, 3015 CN, Netherlands
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Hospital Universitario Virgen del Rocío Pabellón Infantil
RECRUITINGSeville, 41013, Spain
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Hospital de Clínicas de Porto Alegre
RECRUITINGPorto Alegre, Rio Grande do Sul, 90035-003, Brazil
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Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
RECRUITINGBuenos Aires, C1425FNG, Argentina
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New York University Langone Medical Center
TERMINATEDNew York, New York, 10016, United States
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105, United States
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University of Michigan
RECRUITINGAnn Arbor, Michigan, 48109, United States
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University of Utah Medical Center
RECRUITINGSalt Lake City, Utah, 84112, United States
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Universitätsmedizin der Johannes Gutenberg-Universität Mainz
RECRUITINGMainz, 55131, Germany
Conditions
Explore the condition pages connected to this study.