Why Spur Therapeutics’ gene therapy data signals a shift in Gaucher disease bone management
New FLT201 data shows potential bone disease improvement in Gaucher patients. Find out what this means for future treatment strategies.
Pharma & Biotech
Pharma & Biotech
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.
Pharma & Biotech
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.
Pharma & Biotech
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.