The lead drug candidate, ALN1003, showed dose-related body weight loss and favorable body composition changes in the DIO mouse modelALN1003 also ...
PYRUKYND is also approved for adults with PK deficiency in the U.S. and Europe, and a marketing application for PYRUKYND in thalassemia is currently under review by the European Commission. In the U.S ...
Successfully started 216 patients on IBTROZI® (taletrectinib) in the fourth quarter of 2025, for a total of 432 new patient starts since launch in the ...