Effect of nusinersen treatment on quality of life and motor function in adult patients with spinal muscular atrophy
Author:
Publisher
Elsevier BV
Subject
Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health
Reference33 articles.
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2. Copy number variations in the survival motor neuron genes: implications for spinal muscular atrophy and other neurodegenerative diseases;Butchbach;Front Mol Biosci,2016
3. A mouse model for spinal muscular atrophy;Hsieh-Li;Nat Genet,2000
4. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model;Hua;Genes Dev,2010
5. Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3;Maggi;J Neurol Neurosurg Psychiatry,2020
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