Nusinersen helps restore walking ability in childhood spinal muscular atrophy

Pediatr Int. 2019 Jul;61(7):728-729. doi: 10.1111/ped.13867. Epub 2019 Jul 9.

Authors

Mari Sugimoto¹, Kaori Aiba¹, Norihisa Koyama¹, Kenji Yokochi^{1

2}, Hisahide Nishio^{3

4}

Affiliations

¹ Department of Pediatrics, Toyohashi Municipal Hospital, Toyohashi, Aichi, Japan.
² Department of Pediatric Neurology, Seirei-Mikatahara General Hospital, Hamamatsu, Shizuoka, Japan.
³ Department of Community Medicine and Social Healthcare Science, Kobe University Graduate School of Medicine, Kobe, Hyogo, Japan.
⁴ Department of Occupational Therapy, Faculty of Rehabilitation, Kobe Gakuin University, Kobe, Hyogo, Japan.

PMID: 31290219
DOI: 10.1111/ped.13867

No abstract available

Keywords: hybrid gene; magnetic resonance imaging; nusinersen; phenotype; spinal muscular atrophy.

Publication types

Case Reports

MeSH terms

Child, Preschool
Humans
Male
Muscular Atrophy, Spinal / diagnosis
Muscular Atrophy, Spinal / drug therapy*
Muscular Atrophy, Spinal / physiopathology
Neuromuscular Agents / therapeutic use*
Oligonucleotides / therapeutic use*
Walking*

Substances

Neuromuscular Agents
Oligonucleotides
nusinersen