Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study.
Thomas O CrawfordKathryn J SwobodaDarryl C De VivoEnrico Silvio BertiniPaul Wuh-Liang HwuRichard S FinkelJanbernd KirschnerNancy L KuntzAledie Navas NazarioJulie A ParsonsAstrid PechmannMonique M RyanRussell J ButterfieldHaluk A TopalogluTawfeg Ben-OmranValeria Ada SansoneYuh-Jyh JongFrancy ShuCong ZhuStephanie RaynaudTiffany R LagoAngela D ParadisRichard FosterRussell ChinZdenek Bergernull nullPublished in: Muscle & nerve (2023)
These results demonstrate the value of early treatment, durability of treatment effect, and favorable safety profile after ~5 y of nusinersen treatment. Inclusion/exclusion criteria and baseline characteristics should be considered when interpreting presymptomatic SMA trial data.