Gene therapy watch: researchers track Long-Term safety of RGX-111 in MPS i patients
NCT ID NCT06103487
First seen May 04, 2026 · Last updated May 20, 2026 · Updated 2 times
Summary
This study follows 21 people with MPS I who previously received RGX-111 gene therapy in an earlier trial. Researchers will monitor side effects and measure changes in thinking and behavior over time to see if the treatment remains safe and continues to help.
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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 Orange County
Orange, California, 92868, United States
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Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
Conditions
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