The N-Lorem Foundation is gearing up for its Nano-Rare Patient Colloquium 2025 — the third year of its annual event to share the latest developments related to antisense oligonucleotide (ASO) treatment for individuals with extremely rare diseases. The forum, which will take place Oct. 20-21 in Cambridge, Massachusetts,…
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For many children with aromatic l-amino acid decarboxylase (AADC) deficiency, gene therapy holds opportunities for the future. It’s a chance for them to move, speak, and experience life in ways that once seemed impossible. I have often written about the life-changing benefits of gene…
A new analysis makes a case for the value of Kebilidi (eladocagene exuparvovec-tneq), the first gene therapy approved for aromatic L-amino acid decarboxylase (AADC) deficiency. While the one-time treatment has a high cost, it leads to significant improvements in a patient’s quality of life and survival compared to…
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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