OutcomeRx转发了
NEW on the blog: #Kebilidi (eladocagene exuparvovec) just became the first?approved #genetherapy in the U.S.?to be directly administered into the brain. FDA approved Kebilidi for the treatment of aromatic l-amino acid decarboxylase (AADC) deficiency, a #raredisease that causes neurons to produce less dopamine. This contributes to developmental delays, intellectual disabilities, abnormal movements, and autonomic nervous system dysfunction. Read more about the announcement from ASGCT President Paula Cannon, PhD. https://lnkd.in/gRVfcXJb
