Preemptive dual therapy for children at risk for infantile-onset spinal muscular atrophy.
Susan E MatesanzKarlla W BrigattiMillie YoungSabrina W YumKevin A StraussPublished in: Annals of clinical and translational neurology (2024)
Preemptive dual therapy is well tolerated and may provide modest benefit for children at risk for severe spinal muscular atrophy but does not prevent widespread degenerative changes.