Genetics sequencing company Illumina is teaming up with the Center for Data-Driven Discovery in Biomedicine (D3b) on a new project that aims to leverage genomic data from thousands of children to better understand the biological underpinnings of childhood rare diseases and cancers. With this initiative, D3b — a…
First Atom Therapeutics — newly rebranded from Asha Therapeutics — will use a drug design approach powered by artificial intelligence (AI) to develop entirely new therapeutic candidates for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency. The platform will also be used to create novel drugs…
Discussion
There are moments in parenting that feel small when they happen, but later you realize they carry the weight of everything you have been working toward. For me, one of those moments happened at a bowling alley. My daughter, Rylae-Ann, was born with a rare disease called…
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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