My wife, Judy, and I recently traveled back to Taiwan so that our daughter could continue contributing data to her gene therapy clinical trial, and we got to catch up with many of the doctors who have been part of her journey. We also met several families whose children recently…
Discussion
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…
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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