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 Idorsia Pharmaceuticals Ltd’s lucerastat redefine Fabry disease treatment beyond enzyme replacement therapy?
Discover how Idorsia Pharmaceuticals Ltd’s FDA-aligned Phase 3 plan could make lucerastat the first oral therapy for all Fabry disease patients. Read the analysis.