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First Brain-Delivered Gene Therapy Approved for AADC Deficiency
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.
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PTC gets FDA okay for first brain-delivered gene therapy
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 ...