The US Food and Drug Administration (FDA) has granted accelerated approval for PTC Therapeutics’ KEBILIDI, the first gene therapy in the US that is directly delivered to the brain. This 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 ...

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.