SK pharmteco is partnering with Axle Informatics and the National Institutes of Health (NIH) to advance gene therapy programs for rare diseases. The collaboration, which was funded in part by the NIH, focuses on developing viral vectors that use modified viruses to deliver genetic material…
Discussion
Most parents dream about the day their child finally sleeps through the night. When our daughter, Rylae-Ann, finally did, my wife, Judy, and I nearly had heart attacks. Rylae-Ann was born with aromatic l-amino acid decarboxylase deficiency (AADC deficiency), a rare genetic disorder that affects the body’s…
Through a series of long-term experiments using rats, a team of U.S. scientists has tracked how an AAV2-based gene therapy vector similar to the one used in Kebilidi (eladocagene exuparvovec-tneq), an approved gene therapy for AADC deficiency, travels through the brain. Results showed that capsid signals from the…
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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