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.
Genethon advances Duchenne gene therapy trial while licensing Pompe tech to Bayer’s AskBio
Genethon advances gene therapies for Duchenne and Pompe disease. Find out what its nonprofit model and AskBio licensing deal mean for rare disease care.