Researchers have developed a real-time, MRI-guided method to deliver Kebilidi (eladocagene exuparvovec-tneq), an approved gene therapy for people with AADC deficiency, to multiple brain regions, according to a study in nonhuman primates (NHPs). AADC deficiency is characterized by low levels of dopamine and serotonin, two neurotransmitters. While current…
Discussion
There are moments in parenting when you walk into a situation thinking, “This is probably overpriced,” and still go anyway. This was one of those moments. My wife, Judy, and I were looking for another fun adventure. We live in Bangkok, Thailand, where there is no winter…
Aurora Therapeutics has launched a personalized gene-editing platform to develop CRISPR-based therapies — ones that precisely correct disease-causing genetic mutations — for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency. The launch was supported by $16 million in seed funding from Menlo Ventures, a California-based company…
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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