(Q28141072)

11 February 2018

title

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

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11 February 2018

main subject

spinal muscular atrophy

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1 reference

Richard S Finkel

1

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11 February 2018

Claudia A Chiriboga

2

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11 February 2018

Jiri Vajsar

3

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11 February 2018

John W Day

4

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11 February 2018

Jacqueline Montes

5

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11 February 2018

Darryl C De Vivo

6

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11 February 2018

Mason Yamashita

7

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11 February 2018

Frank Rigo

8

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11 February 2018

Gene Hung

9

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11 February 2018

Eugene Schneider

10

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11 February 2018

Daniel A Norris

11

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11 February 2018

Shuting Xia

12

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11 February 2018

C Frank Bennett

13

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11 February 2018

Kathie M Bishop

14

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11 February 2018

language of work or name

English

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publication date

6 December 2016

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11 February 2018

2 references

11 February 2018

volume

388

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11 February 2018

issue

10063

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11 February 2018

page(s)

3017-3026

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11 February 2018

Spinal muscular atrophy: diagnosis and management in a new therapeutic era

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1 reference

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Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens

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Natural history in proximal spinal muscular atrophy. Clinical analysis of 445 patients and suggestions for a modification of existing classifications

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

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Identification and characterization of a spinal muscular atrophy-determining gene

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

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7 January 2021

Correlation between severity and SMN protein level in spinal muscular atrophy

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https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

Consensus Statement for Standard of Care in Spinal Muscular Atrophy

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7 January 2021

Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy

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7 January 2021

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

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7 January 2021

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice

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7 January 2021

Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

1 reference

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7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy

1 reference

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7 January 2021

1 reference

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7 January 2021

Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates

1 reference

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7 January 2021

Prognostic value of the neurologic optimality score at 9 and 18 months in preterm infants born before 31 weeks' gestation.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

Natural history of denervation in SMA: relation to age, SMN2 copy number, and function

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https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

Compound muscle action potential and motor function in children with spinal muscular atrophy

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7 January 2021

Towards harmonisation of outcome measures for DMD and SMA within TREAT-NMD; Report of three expert workshops: TREAT-NMD/ENMC Workshop on outcome measures, 12th–13th May 2007, Naarden, The Netherlands; TREAT-NMD Workshop on outcome measures in exper

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7 January 2021

Development of an ultrasensitive noncompetitive hybridization-ligation enzyme-linked immunosorbent assay for the determination of phosphorothioate oligodeoxynucleotide in plasma

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https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study.

1 reference

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7 January 2021

Characterization of target mRNA reduction through in situ RNA hybridization in multiple organ systems following systemic antisense treatment in animals

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

Genotype-phenotype studies in infantile spinal muscular atrophy (SMA) type I in Germany: implications for clinical trials and genetic counselling

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

The changing natural history of spinal muscular atrophy type 1

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

Spinal muscular atrophy type 1: are proactive respiratory interventions associated with longer survival?

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2816%2931408-8

7 January 2021

10.1016/S0140-6736(16)31408-8

Identifiers

DOI

1 reference

11 February 2018

1 reference

11 February 2018