The Digital Health Measurement Collaborative Community (DATAcc) has launched a core set of digital clinical measures designed to help support the development of new therapies for pediatric rare diseases, such as aromatic l-amino acid decarboxylase (AADC) deficiency. DATAcc is part of the Digital Medicine Society (DiMe), a collaborative group…
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There is a space no one prepares you for. It is not the crisis of diagnosis, nor the triumph of breakthrough treatment. It is something quieter and, in many ways, more complicated. My wife and I felt it most recently while watching our daughter, Rylae-Ann, interact with her cousin, who…
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…
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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