Evolution of bulbar function in spinal muscular atrophy type 1 treated with nusinersen.
Harriet WeststrateGeorgia StimpsonLily ThomasMariacristina ScotoEmily JohnsonAlexandra StewartFrancesco MuntoniGiovanni BaranelloEleanor Conwaynull nullPublished in: Developmental medicine and child neurology (2022)
Impaired bulbar function persisted as a significant complication in most nusinersen-treated patients with SMA1, in contrast to the improvement in motor abilities demonstrated in the majority. p-FOIS allows for tracking of bulbar function progression and treatment response. Larger, prospective studies investigating the longer-term impacts of nusinersen on bulbar function are warranted.