Effect of Nusinersen on Respiratory and Bulbar Function in Children with Spinal Muscular Atrophy: Real-World Experience from a Single Center.
Mirella GaboliMercedes López LobatoJusto Valverde FernándezPatricia Ferrand FerriEloisa Rubio PérezHenry A Andrade RuizJosé María López-Puerta GonzálezMarcos Madruga-GarridoPublished in: Neuropediatrics (2024)
This study provides important insights into the real-world clinical progress of pediatric SMA patients and their response to nusinersen treatment, highlighting the significance of early intervention for better functional and respiratory outcomes.