RARE faces regulatory setback as FDA issues a CRL for UX111, delaying approval of the MPS IIIA gene therapy until 2026.

Ultragenyx's high burn rate, costly R&D, and recent setbacks increase dilution risk, impacting future profitability. Learn ...

This report also provides a detailed analysis of the current mucopolysaccharidosis marketed drugs and late-stage pipeline ...

NHS England has agreed to reimburse Vertex's newest cystic fibrosis medicine. Meanwhile, Merck advanced its once-monthly PrEP ...