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 ...

Q4 2024 Management View CEO Emil Kakkis highlighted 2024 as a pivotal year, with progress in six late-stage programs and a 29% revenue growth over 2023. He confirmed exceeding the upper end of revenue ...

Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...

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.

Pharmaceutical announced new data demonstrating treatment with UX111 AAV gene therapy led to a statistically significant ...