Category: Neuromuscular

Vamorolone for Duchenne Muscular Dystrophy

10 December 2024: The National Institute for Health and Care Excellence (NICE) has approved vamorolone (Agamree, Santhera) as a treatment option for Duchenne Muscular Dystrophy (DMD) in individuals aged 4 years and over, following a revised pricing agreement with the manufacturer. Despite initial rejection in March due to uncertainties in evidence and cost-effectiveness, further analyses…