What Is Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency? AADC deficiency is an ultra-rare genetic disorder. It affects how your cells send signals to each other. People born with this condition ...
Kebillidi is the first gene therapy to gain FDA approval for the treatment of aromatic L-amino acid decarboxylase deficiency. The FDA has approved PTC Therapeutics’ Kebillidi (eladocagene ...
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