共 207 条
- [1] Kolb SJ(2017)Natural history of infantile-onset spinal muscular atrophy Ann Neurol 82 883-891
- [2] Coffey CS(2018)Muscle strength and motor function throughout life in a cross-sectional cohort of 180 patients with spinal muscular atrophy types 1c-4 Eur J Neurol 25 512-518
- [3] Yankey JW(2017)218th ENMC International Workshop: revisiting the consensus on standards of care in SMA Naarden, The Netherlands, 19-21 February 2016 Neuromuscul Disord 27 596-605
- [4] Krosschell K(2018)Diagnosis and management of spinal muscular atrophy: part 1: recommendations for diagnosis, rehabilitation, orthopedic and nutritional care Neuromuscul Disord 28 103-115
- [5] Arnold WD(2018)Diagnosis and management of spinal muscular atrophy: part 2: pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics Neuromuscul Disord 28 197-207
- [6] Rutkove SB(2017)Nusinersen versus sham control in infantile-onset spinal muscular atrophy N Engl J Med 377 1723-1732
- [7] Swoboda KJ(2015)A large animal model of spinal muscular atrophy and correction of phenotype Ann Neurol 77 399-4147
- [8] Reyna SP(2017)FDA-approved oligonucleotide therapies in 2017 Mol Ther 25 1069-1075
- [9] Sakonju A(2018)Single-center experience with intrathecal administration of Nusinersen in children with spinal muscular atrophy type 1 Eur J Paediatr Neurol 22 122-127
- [10] Darras BT(2017)Expanded access program with Nusinersen in SMA type I in Italy: strengths and pitfalls of a successful experience Neuromuscul Disord 27 1084-1086