Ultragenyx Pharmaceutical (RARE) announced that it has reached alignment with the FDA regarding the phase III study design and endpoints for GTX-102, an antisense oligonucleotide for Angelman syndrome, following the completion of a successful end-of-phase II meeting with the regulatory body. The company expects to initiate the same by the end of 2024.In the meeting, Ultragenyx mainly discussed the positive interim data from the phase I/II study, announced in April 2024, with the FDA. The study evaluated the ...