This study focuses on patients with a neurodevelopmental disorder caused by changes in the CTNNB1 gene. This is a rare condition that affects brain development and movement abilities. The study will test a treatment combining two medications: levodopa and carbidopa, which are taken as oral capsules.
The purpose of this research is to determine if this treatment can improve movement abilities in people with CTNNB1-related neurodevelopmental disorder. The treatment involves taking specific doses of levodopa and carbidopa that are calculated based on each person’s weight. The study will last for 12 months, during which participants will take these medications daily.
Throughout the study, doctors will monitor how well patients can move and perform various physical activities. They will also track changes in thinking abilities, language development, and overall quality of life. The safety of the treatment will be carefully watched through regular medical check-ups and by keeping track of any side effects that may occur.



France