For some families, this is a daily reality due to rare genetic disorders. One such condition is Sanfilippo syndrome, which creates significant hurdles for affected children and their families.
Goldman Sachs reiterated its Buy rating on Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares with a steady price target of $78.00. The firm's analyst highlighted Ultragenyx's fourth-quarter total ...
Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...
Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...