A new gene therapy has been given an accelerated approval in the US for an inherited disorder affecting the way neurons transmit information to other cells in the body. The therapy, Kebilidi (eladocagene exuparvovec), has been shown to be effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency, a rare disease. It is caused by mutations in the DDC gene leading to shortages of the AADC enzyme which plays an important role in the nervous system.
