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) ...
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 ...
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 ...
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 ...
More than two years after its approval in Europe, PTC Therapeutics' gene therapy for rare genetic disorder AADC deficiency will soon be made available to patients in the US. The FDA has approved PTC's ...
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