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 AB-1009 reshape treatment for late-onset Pompe? AskBio prepares for first patient in U.S.
AskBio’s AB-1009 gene therapy for late-onset Pompe secures FDA IND clearance. Find out what this milestone means for AAV innovation and patient outlook.