Kebilidi (eladocagene exuparvovec-tneq), an approved gene therapy delivered directly to the brain to treat AADC deficiency, led to clinical benefits even when only a small portion of the targeted brain region was reached by the treatment, according to a small study in France. Clinical benefits were…
Discussion
Our daughter’s journey has never been a straight path, and if there is one thing my wife, Judy, and I have learned, it is to always have a full tank of adaptability and perseverance. Rylae-Ann was born with aromatic l-amino acid decarboxylase (AADC) deficiency, a rare neurological…
Discussion
Even before our daughter, Rylae-Ann, was diagnosed with aromatic l-amino acid decarboxylase (AADC) deficiency, my wife, Judy, began taking meticulous notes. We were not writing a book. We were just trying to understand what was happening to our family. AADC deficiency is an ultra-rare genetic disorder that…
Aromatic l-amino acid decarboxylase (AADC) deficiency is a genetic and neurometabolic disease that affects the nervous system. Learn more about the disease here.
AADC deficiency can affect many aspects of a person’s life, including mobility, breathing, sleeping, and eating. Learn more here about different ways to manage the condition, adapt, and handle everyday tasks.
There is not yet a cure for AADC deficiency, but there are approved treatments that can help manage the condition, as well as experimental treatments in the pipeline. Learn more about treating the disease here.
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