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1st gene therapy delivered directly into brain approved
Becker's Healthcare November 20, 2024
The FDA granted accelerated approved to the first gene therapy directly administered into the brain on Nov. 14.
The therapy, Kebilidi, was approved to treat adults and children with aromatic L-amino acid decarboxylase deficiency, a rare genetic disorder that affects the body’s ability to produce neurotransmitters that support motor function.
Kebilidi is administered via four infusions into a part of the brain...
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