"In July this year, Upstaza became the first approved disease-modifying treatment for AADC deficiency and the first marketed gene therapy directly infused into the brain when it was authorized by the European Medicines Agency."
In Parkinson's cases where there is a genetic problem, this is encouraging.
AADC deficiency is an ultra-rare, inherited genetic disease which typically manifests within the first year of life. It is caused by changes in the gene that produces the AADC enzyme which is needed to produce certain substances vital for the normal functioning of the brain and nerves, including dopamine and serotonin. These substances are used by cells in the brain and the nervous system to send signals and are crucial for the development of motor functions.
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