The California Institute for Regenerative Medicine (CIRM) approved a $100 million funding program designed to speed the development of gene therapies for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency. The Rare Disease Acceleration Platform and Innovation and Delivery (RAPID) program will invest the funds over two years…
Discussion
For our family, the Chinese New Year has always been more than just a date on the calendar. It’s a reminder for us to pause, reflect, clear out the clutter — both literal and emotional — and move forward with hope. My wife, Judy, is from Taiwan, so these…
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…
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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