Experimental Treatments for AADC

Overview

While current treatments can help manage symptoms of aromatic L-amino acid decarboxylase (AADC) deficiency, researchers are exploring new therapeutic approaches designed to target the underlying causes of this genetic and neurometabolic disorder.

VGN-RO9b

VGN-R09b is an investigational gene therapy designed to deliver two genes: a working copy of the DDC gene, which encodes the AADC enzyme, and a second gene that encodes a neurotrophic factor that supports nerve cell health. The therapy aims to restore dopamine production and improve neurotransmitter signaling affected by AADC deficiency.

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