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…
Discussion
There were many times when my wife, Judy, and I almost lost our daughter, Rylae-Ann. The early years of her life were filled with medical emergencies, unanswered questions, and moments when the future felt painfully uncertain. Aromatic l-amino acid decarboxylase (AADC) deficiency is not a journey anyone…
Over the course of 2025, U.S.-based Genedx published dozens of studies powered by its rare disease genomics dataset — which, by the numbers, the testing company claims, shows the effectiveness of a genome-first approach. Genomics is the study of an organism’s entire genome, essentially all of its DNA. This…
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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