Ultragenyx Pharmaceutical Inc. (RARE) Presents at Leerink Global Healthcare Conference 2026 Transcript

Commercial Success - The company has experienced significant commercial success driven by Crysvita, showing double-digit growth year-over-year [1] - Other products such as Dojolvi and Evkeeza are also contributing to this growth, with Mepsevii providing additional support [1] Pipeline Development - A major focus is on the Angelman syndrome program, with data readout expected in the second half of the year [2] - Progress is being made in the gene therapy pipeline, with a PDUFA date for GSDIa and Sanfilippo nearing completion [2] - The company is also advancing other projects related to OTC and Wilson disease, indicating a robust clinical pipeline [2]