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Evolution of bulbar function in spinal muscular atrophy type 1 treated with nusinersen.

Harriet WeststrateGeorgia StimpsonLily ThomasMariacristina ScotoEmily JohnsonAlexandra StewartFrancesco MuntoniGiovanni BaranelloEleanor Conwaynull null
Published 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.
Keyphrases
  • magnetic resonance
  • preterm infants
  • magnetic resonance imaging
  • newly diagnosed