共 68 条
[1]
Yeo CJJ(2020)Overturning the paradigm of spinal muscular atrophy as just a motor neuron disease Pediatr Neurol 86 890-897
[2]
Darras BT(2016)Results from a phase 1 study of nusinersen (ISIS-SMN (Rx)) in children with spinal muscular atrophy Neurology 388 3017-3026
[3]
Chiriboga CA(2016)Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study Lancet Lond Engl 377 1723-1732
[4]
Swoboda KJ(2017)Nusinersen versus sham control in infantile-onset spinal muscular atrophy N Engl J Med 378 625-635
[5]
Darras BT(2018)Nusinersen versus sham control in later-onset spinal muscular atrophy N Engl J Med 15 148-127
[6]
Iannaccone ST(2020)Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study Orphanet J Rare Dis 22 122-e1318
[7]
Montes J(2018)Single-center experience with intrathecal administration of Nusinersen in children with spinal muscular atrophy type 1 Eur J Paediatr Neurol EJPN 91 e1312-933
[8]
De Vivo DC(2018)Nusinersen in patients older than 7 months with spinal muscular atrophy type 1: a cohort study Neurology 91 923-24
[9]
Finkel RS(2018)Evidence in focus: nusinersen use in spinal muscular atrophy Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology Neurology 55 18-5
[10]
Chiriboga CA(2019)Putting our best foot forward: clinical, treatment-based and ethical considerations of nusinersen therapy in Canada for spinal muscular atrophy J Paediatr Child Health 5 3-146