Researchers at the Keck School of Medicine, University of Southern California, are receiving up to $6.8 million in funding to develop an artificial intelligence (AI)-based computational model to help advance gene and cell therapies for children with rare diseases, including conditions such as aromatic L-amino acid decarboxylase (AADC) deficiency. Funded…
Discussion
When NASA launches a rocket, most people watch for a few minutes, feel a sense of awe, and move on with their day. My wife, Judy, saw a week of lessons. That is the difference between traditional schooling and intentional learning. One passes by moments. The other builds entire experiences…
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…
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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