PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...
I just felt nervous at the thought of having to deal with a feeding tube or something. I was living in a two-bedroom condo ... James’s pediatrician agreed that James wasn’t hitting his milestones and ...
First-ever FDA approval for gene therapy directly administered to the brain - - Priority review voucher granted - - Broad label including children and adults - - ...