The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...

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 ...

The study portrays the intense need for daily care experienced by children with aromatic L-Amino acid decarboxylase deficiency (AADC-d) and their families. Parents or caregivers of children with the ...

AADC deficiency is a rare and fatal genetic disorder that typically causes severe disability starting during the first months of life. Children living with AADC deficiency can experience seizure-like ...

This new analysis of 5-year data on a novel gene therapy from PTC Therapeutics shows both cognitive and motor function improvement in children with aromatic L-amino acid decarboxylase (AADC) ...

“This is nothing short of a miracle," mom Shante Stagg told TODAY of a gene therapy that has helped her son who has a rare health condition. After a complicated birth, Jamell Stagg Jr.’s parents ...

The FDA granted accelerated approval to eladocagene exuparvovec (Kebilidi) to treat children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency, the agency announced Thursday. The ...

Five-year-old Boston Yannotti’s ear-to-ear smile masks what has been an extremely difficult childhood. The young boy, with reddish-brown hair and bright blue eyes like his mother, has been confined ...