Gene therapy study for adults with MPS type I
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This study tests a new gene therapy called JWK008 for adults with MPS type I. The goal is to see if it is safe and can help improve symptoms. You may be able to join if you are 18 or older and have not had serious side effects from enzyme replacement therapy.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 18 years or older.
- You have been diagnosed with MPS type I (a rare genetic condition).
- You have not had an allergic reaction to enzyme replacement therapy (like laronidase).
- You are willing to use reliable birth control if you or your partner could become pregnant.
- Your liver MRI shows no masses (a normal scan).
- You have not had gene therapy before and are not currently in another clinical trial.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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