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FDA designates Ionis Breakthrough Therapy for Angelman syndrome after Phase 1/2 results showed functional improvements.
HC Wainwright & Co. analyst Raghuram Selvaraju reiterates Ultragenyx Pharmaceutical (NASDAQ:RARE) with a Buy and maintai...
At Week 96 participants experienced even greater reductions in daily cornstarch intake while maintaining low levels of hypoglyc...
Cantor Fitzgerald analyst Kristen Kluska reiterates Ultragenyx Pharmaceutical (NASDAQ:RARE) with a Overweight and maintains ...
FDA launches Rare Disease Evidence Principles to guide therapy reviews, offering new pathways for approval while raising questi...
Canaccord Genuity analyst Whitney Ijem maintains Ultragenyx Pharmaceutical (NASDAQ:RARE) with a Buy and lowers the price tar...