What effect does AADC have on children?

Marcus Allen
Marcus Allen

AADC is a very rare form of Parkinson’s. Symptoms include; developmental delays, weak muscle tone, muscle stiffness and involuntary writhing movements of the limbs.

Around 100 children suffer from Aromatic Amino Acid Decarboxylase Deficiency (AADC) worldwide, with only a handful being based in the UK. Unfortunately, a cure has not yet been found for AADC, however, there are two treatments for the condition.

The treatments are rarely funded by the British National Health Service (NHS), and so, Many children like Marcus Allen have to try to raise the money to get life changing treatments.

Marcus Allen is a 12 year old boy living in Stockport, UK. He suffers from the rare form of Parkinson’s disease, AADC. Marcus’ family are currently trying to raise enough money in order to pay for a special type of gene therapy in Poland. The therapy costs around €79,000 or £70,000. The NHS have said that they will not cover the surgery, so his family will have to raise the money privately. There have been a variety of successes in the AAV2-hAADC vector treatment, with the effects radically changing the quality of life of those suffering from AADC.

According to the AADC Research Trust: “In order for the AAV2-hAADC vector to be delivered to the correct brain cells it needs to be directly injected into a specific region of the brain. Professor Krystof Bankiewicz and his team have developed pioneering surgical techniques to ensure accurate delivery of a vector to the selected brain region. The system uses a specially designed cannula that is inserted into the brain to allow direct infusion of a liquid containing the AAV2-hAADC vector. The patient’s head is placed in a specialised stereotactic frame and cannula guide devices are attached. Real-time MRI imaging of the patient’s brain is used to map the target region, plan cannula insertion and positioning, and calculate how much vector to infuse.”

How can you help?

Any donation will help Marcus take one step closer to receiving a treatment that will absolutely change his life. To donate click here.

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