13th RWE, Market Access, Pricing & Reimbursement 2026 Europe

Gene therapy reimbursement in the Netherlands has demonstrated clear clinical value since the 2021 conditional access agreement for voretigene neparvovec (Luxturna®). Five years of structured real-world use confirmed that carefully designed protocols successfully selected appropriate patients wit...

Novartis has achieved a key clinical milestone with delpacibart braxlosiran FSHD, as its FORTITUDE biomarker cohort successfully met the primary endpoint by substantially suppressing DUX4-driven gene expression and reducing muscle damage markers in patients with facioscapulohumeral muscular dystr...

Athlete risk stratification now integrates epidemiological evidence, explainable artificial intelligence, and mechanistic cardiac models to better protect young competitors. This convergence offers a path to distinguish healthy athletic remodeling from hidden substrates that can trigger fatal arr...