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.

Reports Q4 revenue $164.88M, consensus $158.71M. “We have created a next-generation rare disease company on a pathway to profitability with ...

GC Biopharma, a South Korean biopharmaceutical company, announced on Feb. 10th that it presented development updates on its ...

Ultragenyx Pharmaceutical Inc (RARE) reports a 29% revenue increase in 2024, driven by successful product launches and international expansion, despite a significant net loss.

The Grand Rapids Police Department will face the Kentwood Police Department in just one of the matchups scheduled for the ...

Ultragenyx Pharmaceutical RARE reported new findings from an ongoing, pivotal study of its investigational candidate, UX111 (ABO-102) AAV gene therapy, for Sanfilippo syndrome type A (MPS IIIA).