Parkinson's disease is a disorder of the brain that leads to shaking (tremors) and difficulty with walking, movement and coordination. It happens when the brain doesn't produce enough of a chemical called dopamine.
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Our Movement Disorder Program focuses on Parkinson’s and other movement disorders. Providing a swift, precise diagnosis, and treatment to optimize your quality of life.
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The Neurorehabilitation program offers diagnosis and treatment for people with cerebral palsy, multiple sclerosis, Parkinson’s disease, brain injuries and spinal cord injuries.
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Research + Clinical Trials
The main purpose of this study is to test the safety and efficacy (how well it works) of an investigational product called VY-AADC02 on the severity of PD. VY-AADC02 involves placing a gene in the brain that programs brain cells to produce an enzyme (protein) called AADC (aromatic amino acid decarboxylase).
Genes are what make up our genetic code, aka DNA – the chemical structure carrying all the information that determines many human traits and characteristics such as the color of your eyes or hair. Each gene can be read by your cells (the building blocks of the human body) as a set of instructions to make a specific protein.
AADC is a naturally occurring protein found in the brain that converts levodopa (one of your current PD medications) to dopamine. Dopamine is a chemical “messenger” that allows the brain cells to communicate with each other and is involved with the normal function of movement control centers in the brain. As PD worsens, the amount of AADC in the brain decreases, and, in turn, this decrease reduces the amount of dopamine that is produced from each dose of levodopa that you take.
In order to deliver the AADC gene into the brain cells, the gene is delivered inside a transporter called adeno-associated viral vector (AAV). To explain this, think about the AADC gene like a letter that carries the information that the brain will read. The AAV acts as the envelope that contains this information, and the combined AADC and AAV can be thought of as the complete package (the letter and envelope that the brain will receive). AAV is used because it is not known to cause disease or spread to others. The combined package of AADC and AAV will be referred to as the “study product” in the remainder of this document. The study product will tell the brain cells to make more AADC, which may increase dopamine levels in your brain when you take levodopa. An increase in dopamine levels may provide improvement in the movement areas in the brain and may help with your PD symptoms.
The investigators hope to learn more about the delivery and distribution of the study product, including the delivery device used in the surgery (called a cannula), the imaging substance (gadolinium) used to visualize the infusion of the study product during the surgery, and how the body reacts to the study product over time using PET (positron emission tomography) scans to determine the change in the level of the AADC gene in your brain. Gadolinium is routinely used for the purpose of imaging, in this study it is considered experimental as it is infused directly into the tissue of the brain, and not through a vein.
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The aim of this study is to test the safety and efficacy of IPX203 compared to IR CD-LD in participants with Parkinson’s disease. IPX203 is an investigational extended-release (i.e. releases drug more slowly) capsule formulation of carbidopa-levodopa (CD-LD) administered orally (by mouth).
IPX203 is being investigated to determine whether the drug is safe and potentially has a better effect than currently approved IR CD-LD. If successful, the drug could possibly improve the daily control of motor symptoms in people with Parkinson’s disease.
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