Six and a half years ago, my wife, Judy, and I placed our trust in a medical team in Taiwan and handed our daughter, Rylae-Ann, over for a surgery we hoped would change her future. Today, we continue returning to Taiwan, not because Rylae-Ann needs another operation, but because she…
Discussion
The N-Lorem Foundation has partnered with Servier to develop novel antisense oligonucleotide (ASO) therapeutic candidates for people with extremely rare neurodevelopmental disorders. The collaboration aims to expand the number of patients with nano-rare neurological disorders that N-Lorem can treat. Under the agreement, N-Lorem will use its ASO technology platform to create…
Discussion
The U.S. Food and Drug Administration (FDA) has issued draft guidance meant to help developers of human gene therapies involving genome editing bring treatments to patients more efficiently, including those with rare and life-threatening diseases. Some recommendations also may apply to other cell and gene therapy products,…
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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