Risdiplam tablet formulation becomes available in Italy for SMA patients

A new tablet formulation of risdiplam is now available in Italy for the treatment of Spinal Muscular Atrophy (SMA). This oral medication joins the existing syrup solution, offering increased flexibility for patients to manage their daily lives, work, and studies without the logistical constraints of previous administration methods.

During a Roche Italia event in Milan, Stefania Pedroni, president of the Italian Union for the Fight against Muscular Dystrophy (Uildm), emphasized that while new therapies have improved life expectancy and quality, neonatal screening remains a critical unmet need. She noted that ensuring consistent screening across all Italian regions is essential for early intervention.

Neurologist Lorenzo Maggi from the Carlo Besta Neurological Institute highlighted that the tablet formulation improves therapeutic adherence and supports personalized medicine. He noted that innovative therapies have radically changed the natural history of the disease, particularly for severe cases like SMA1, which were previously often fatal.