Global effort launched to map rare genetic disease

NCT ID NCT03604835

Recruiting now Knowledge-focused Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

Summary

This study aims to better understand how MPS VII (Sly Syndrome) progresses and how the treatment vestronidase alfa works over the long term. It will follow about 50 patients, both those receiving the treatment and those who are not, to collect detailed health information. The goal is to build a comprehensive picture of the disease and the treatment's safety and effectiveness.

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

Contact

Phone: •••-•••-•••• Email: •••••@•••••
Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Centre Hospitalier Universitaire La Timone
RECRUITING

Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France
Centro Hospitalar do Porto
RECRUITING

Porto, 4050-651, Portugal
Children's Hospital of Orange County
RECRUITING

Orange, California, 92868, United States
Children's National Health System
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Erasmus University Medical Center Rotterdam
TERMINATED

Rotterdam, South Holland, 3015 CN, Netherlands
Hospital Universitario Virgen del Rocío Pabellón Infantil
RECRUITING

Seville, 41013, Spain
Hospital de Clínicas de Porto Alegre
RECRUITING

Porto Alegre, Rio Grande do Sul, 90035-003, Brazil
Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L.
RECRUITING

Buenos Aires, C1425FNG, Argentina
New York University Langone Medical Center
TERMINATED

New York, New York, 10016, United States
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98105, United States
University of Michigan
RECRUITING

Ann Arbor, Michigan, 48109, United States
University of Utah Medical Center
RECRUITING

Salt Lake City, Utah, 84112, United States
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
RECRUITING

Mainz, 55131, Germany

Conditions

Explore the condition pages connected to this study.

MPS 7 MPS VII MUCOPOLYSACCHARIDOSIS VII SLY SYNDROME