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.

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

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

WORLD Symposium 2025 is an international conference or lysosomal storage disorders experts to share and exchange insights for researching better treatment of the disorders. At thi ...

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