Hope for hunter syndrome: new drug shows promise in Long-Term trial
NCT ID NCT05594992
First seen Oct 31, 2025 · Last updated May 14, 2026 · Updated 26 times
Summary
This study looks at the long-term safety and effectiveness of a drug called JR-141 for people with Hunter syndrome, a rare genetic disorder. About 80 participants who completed an earlier study will continue treatment. Researchers will track changes in brain function, thinking skills, and physical abilities over several years.
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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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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.