Oral Pompe therapy S-606001 moves to Phase 2 as Shionogi expands rare disease focus
Shionogi’s S-606001 enters global Phase 2 as the first oral GYS1 inhibitor tested as an add-on to ERT in late-onset Pompe disease. Read the analysis.
Can KalVista’s refill-led EKTERLY growth signal a new standard in on-demand HAE care?
KalVista’s oral HAE drug EKTERLY hit $49M in 2025 sales. Discover what strong refill demand and global expansion mean for rare disease treatment models.