One-time treatment with Kebilidi (eladocagene exuparvovec-tneq) helped children with aromatic L-amino acid decarboxylase (AADC) deficiency gain new motor abilities within about a year, according to data from an ongoing clinical trial. The findings come from a Phase 2 clinical trial (NCT04903288) testing Kebilidi in 13 children with…
Discussion
There was a stretch of time when I genuinely thought I was going insane. Night after night, the crying came in waves that instantly snapped my wife, Judy, and me from our sleep, hearts racing, and already exhausted from the 24-hour care we provided during the day. Our daughter,…
A boy with a severe form of aromatic L-amino acid decarboxylase (AADC) deficiency was able to walk independently — and later run — after receiving the gene therapy Kebilidi (eladocagene exuparvovec), according to a new case report. The child, now 5, showed rapid and sustained improvements in movement…
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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