Two U.S.-based companies are teaming up to advance personalized human-based disease models that could help speed the discovery of novel therapies for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency. Ixcells Biotechnologies and Rosebud Biosciences, both headquartered in California, have entered into a partnership to develop…
Discussion
My wife, Judy, and I have always been a competitive couple. Backgammon? Intense. Canasta? Ruthless. Ping-Pong? Fast-paced! Trash talk was common, laughter was loud, and someone usually demanded a rematch. Naturally, when our daughter, Rylae-Ann, was born, we assumed she’d be drafted straight into family game night. Then life dealt…
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…
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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