Earlier this year, the FDA approved 2 new indications for Aquaflor. According to Merck, the supplemental approval increased ...
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidiâ„¢ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...
This is the first gene therapy for AADC deficiency to receive US regulatory approval and the first that can be administered directly to the brain.