Better treatments for Gaucher disease with lung or bone problems
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study looks at how standard treatments for Gaucher disease affect lung problems, bone damage, and parkinson-like symptoms. It aims to find better ways to treat these complications and improve quality of life.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have Gaucher disease type 2 or 3, confirmed by genetic testing
- You have signs of lung disease or swollen lymph nodes in the chest or belly
- You are currently getting treatment or are eligible to start standard treatment
- You are at least 3 months old
- You can agree to take part (or a parent or guardian can)
- For the bone study: you are between 10 and 75 years old and have ongoing bone problems despite long-term treatment
- For the Parkinson study: you have Gaucher disease and symptoms of Parkinson disease
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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