Long-term nusinersen treatment across a wide spectrum of spinal muscular atrophy severity: a real-world experience.
Anna ŁusakowskaAdrianna WójcikAnna FrączekKarolina Aragon-GawińskaAnna Potulska-ChromikPaweł BaranowskiRyszard NowakGrzegorz RosiakKrzysztof MilczarekDariusz KoneckiZuzanna Gierlak-WójcickaMałgorzata BurlewiczMaria JędrzejowskaPublished in: Orphanet journal of rare diseases (2023)
In our study, nusinersen led to continuous functional improvement over 30-month follow-up and was well tolerated by adults and older children with a wide spectrum of SMA severity.