The Canadian government has signed a bilateral agreement with Saskatchewan, a province in Western Canada, to improve access to treatments for rare diseases and support enhanced access to existing drugs, early diagnosis, and screening for rare diseases, such as aromatic l-amino acid decarboxylase (AADC) deficiency. The agreement includes an…
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For our family, 2024 was a year of milestones and overcoming challenges. It was a good year, and progress was OK. But ever since our daughter, Rylae-Ann, was diagnosed with the rare disease aromatic l-amino acid decarboxylase (AADC) deficiency, “good” and “OK” have not been enough for…
The N-Lorem Foundation has partnered with Gondolabio to discover and advance novel antisense oligonucleotide (ASO) treatment options — medications that can correct genetic abnormalities in people — for nano-rare patients, or those with extremely rare diseases. First, the California-based nonprofit will leverage its ASO discovery platform to identify…
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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