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.
Why Beren Therapeutics’ adrabetadex NDA could redefine approval standards in ultra-rare pediatric disease
FDA acceptance of adrabetadex puts disease modification for infantile-onset NPC under review. See what this decision could change for rare disease approvals.
Is JCR unlocking the CNS for lysosomal storage diseases before gene therapy gets there?
Find out how JCR Pharmaceuticals is redefining lysosomal disease therapy with brain-targeted delivery platforms.