One-Shot gene therapy aims to treat devastating brain disease
NCT ID NCT03580083
Summary
This early-stage study is testing a one-time gene therapy called RGX-111 for people with MPS I, a rare genetic disease that damages the brain. The therapy is injected into the fluid around the brain and spine to deliver a working gene, hoping to help the brain produce a missing enzyme. The main goal is to see if the treatment is safe and well-tolerated, while also checking if it improves thinking, learning, and daily living skills over two years.
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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
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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