Hope for hunter syndrome: Long-Term brain treatment study continues
NCT ID NCT05594992
Summary
This study continues testing JR-141 to see if it remains safe and effective for Hunter syndrome patients over the long term. It follows 80 patients who were already in the main study, tracking how their thinking skills, walking ability, and organ health change. The goal is to understand if this treatment can continue helping control this rare genetic disease.
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Contacts and locations
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Hospital Sant Joan de Déu
Barcelona, Spain
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Hospital Universitario Austral
Buenos Aires, Argentina
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Hospital de Clínicas de Porto Alegre
Porto Alegre, Brazil
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Hôpital Femme Mère Enfant
Bron, France
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Instituto de Genética e Erros Inatos do Metabolismo
São Paulo, Brazil
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Osp. Pediatrico Bambino Gesù, IRCCS
Rome, Italy
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
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Uniwersytecki Szpital Dziecięcy
Krakow, Poland
Conditions
Explore the condition pages connected to this study.