Cure SMA and our patient community celebrate the first approved drug for SMA.

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Targeting Amyloid-β Precursor Protein, APP, Splicing with Antisense Oligonucleotides Reduces Toxic Amyloid-β Production.Mechanism of Splicing Regulation of Spinal Muscular Atrophy Genes.

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Cure SMA and our patient community celebrate the first approved drug for SMA.

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2017 nî lūn-bûn

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2017年の論文

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2017年学术文章

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2017年学术文章

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2017年学术文章

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2017年學術文章

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Cure SMA and our patient community celebrate the first approved drug for SMA.

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Cure SMA and our patient community celebrate the first approved drug for SMA.

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Cure SMA and our patient community celebrate the first approved drug for SMA.

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Cure SMA and our patient community celebrate the first approved drug for SMA.

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Cure SMA and our patient community celebrate the first approved drug for SMA.

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Cure SMA and our patient community celebrate the first approved drug for SMA.

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P1476

Cure SMA and our patient community celebrate the first approved drug for SMA.

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P2093

J Glascock

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J Jarecki

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K Hobby

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M Lenz

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P2860

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.

Identification and characterization of a spinal muscular atrophy-determining gene

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.

Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.

Emerging therapies and challenges in spinal muscular atrophy

Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

Observational study of spinal muscular atrophy type I and implications for clinical trials.

Temporal requirement for high SMN expression in SMA mice

Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron.

P2888

gt.2017.39

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scholarly article

P356

10.1038/GT.2017.39

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2017-05-15T00:00:00Z

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28504658

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Cure SMA and our patient community celebrate the first approved drug for SMA.

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P2860

P2860

Cure SMA and our patient community celebrate the first approved drug for SMA.

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