Shionogi has entered into an agreement to acquire IntraBio, the company behind AQNEURSA® (levacetylleucine). Here is what the announcement means—and what we are still waiting to learn—for the Australian NPC community.

Cyclo Therapeutics has shared topline results from its Phase 3 TransportNPC study, which included Australian participants. While the study’s main endpoint was not statistically significant, important findings—including a reported survival benefit for children with infantile-onset NPC—warrant close attention and further review.