Medications to Avoid in AADC Deficiency

Emily Malcolm, PhD avatar

by Emily Malcolm, PhD |

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lumbar puncture and AADC deficiency

Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare genetic disease that affects the nervous system.

How do doctors treat AADC deficiency?

AADC deficiency is very rare, with only about 100 people with the disease described in studies. This means that it is very difficult for doctors to predict whether available treatments will be of help to some patients, all patients, or only a few patients. It is also difficult to predict the effect that medications you may use for other problems will have on this disease.

Medications used for  AADC deficiency are aimed at increasing dopamine and serotonin in the brain. These are two important neurotransmitters (cell signaling molecules in the brain) that people with AADC deficiency cannot make enough of.

Medications that block the actions of dopamine or interact with serotonin receptors should be avoided by people with AADC deficiency.

Dopamine antagonists

Dopamine antagonists work by blocking dopamine receptors, making fewer receptors available to bind to dopamine. These medications can make the symptoms of AADC deficiency worse and should be avoided.

Haloperidol (an antipsychotic medication) and metoclopramide (an anti-nausea medication) are two examples of dopamine antagonists that you should not give to someone with AADC deficiency.


Certain types of anti-emetics, medications intended to help with nausea or vomiting like ondansetron, and granisetron, are also not suitable because they interact with serotonin receptors. Although there is no clinical data as yet, these medications may cause severe side effects in AADC deficiency patients, because these people do not make enough serotonin.

Keeping a record of your child’s medication

It’s important to discuss your child’s medications with your doctor before making any changes. Because AADC deficiency is a rare disease, not all medical professionals may be aware of its symptoms and causes.

It’s also a good idea to specify those medications that your child has not responded to (or has responded poorly to) in these records, in case of an emergency or if your child must be treated by someone other than their primary care physician.


Last updated: April 28, 2020.


AADC News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified healthcare providers with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.