NHS Drugs Go Ahead Offers Lifeline to Children with Rare Muscle Wasting Disease The National Institute for Health and Care Excellence (Nice) has issued guidance paving the way for hundreds of children in England, Wales, and Northern Ireland to receive treatment for spinal muscular atrophy (SMA).
This debilitating condition causes severe muscle weakness, affecting mobility, breathing, and swallowing.
Without treatment, patients face a bleak prognosis, with those suffering from the most severe form typically dying before their second birthday.