Publications by authors named "Misuzu Hoshino"
Purpose: Nusinersen is an antisense oligonucleotide approved for the treatment of spinal muscular atrophy (SMA). A post-marketing surveillance (PMS) has been ongoing (August 2017-August 2025) in all patients in Japan who were administered nusinersen intrathecally in real-world clinical settings. We report the interim analysis results for safety and effectiveness.
View Article and Find Full Text PDFNusinersen is an antisense oligonucleotide for the treatment of spinal muscular atrophy (SMA). A post-marketing surveillance (PMS) has been ongoing (August 2017-August 2025) in all patients in Japan who received intrathecal nusinersen in real-world clinical settings. We report the interim analysis results of safety and effectiveness.
View Article and Find Full Text PDFArticle Synopsis
- Nusinersen is the first therapy that modifies the disease progression of spinal muscular atrophy (SMA), and this report reviews its safety and effectiveness in Japanese patients using two data sources.
- The analysis includes data from an ongoing post-marketing surveillance study and Biogen’s safety database, covering treatments from August 2017 to mid-2019, assessing adverse events and effectiveness through motor function improvements.
- Of the 271 patients studied, around 25% experienced adverse events, while over 26% showed improvements in motor function, indicating that nusinersen has a favorable benefit-risk profile for treating SMA in Japan.