Gene therapy for Type 1 Gaucher disease
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests a new gene therapy called LY-M001 for adults with Type 1 Gaucher disease. It aims to help the body produce the missing enzyme and reduce symptoms like low blood counts or enlarged organs.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 60 years old.
- You have been diagnosed with Type 1 Gaucher disease through genetic testing and a blood test showing very low enzyme activity.
- You are either currently on stable treatment for Gaucher disease, or if untreated, you have low blood counts, an enlarged liver, or an enlarged spleen.
- You have not received any gene therapy before.
- You do not have serious liver disease, active hepatitis B or C, or a history of cancer in the last 5 years.
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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