(Q45855674)

17 December 2017

title

Eteplirsen for the treatment of Duchenne muscular dystrophy (English)

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17 December 2017

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14

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17 December 2017

author name string

Jerry R Mendell

1

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17 December 2017

Louise R Rodino-Klapac

2

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17 December 2017

Zarife Sahenk

3

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17 December 2017

Kandice Roush

4

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17 December 2017

Loren Bird

5

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17 December 2017

Linda P Lowes

6

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17 December 2017

Lindsay Alfano

7

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17 December 2017

Ann Maria Gomez

8

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17 December 2017

Sarah Lewis

9

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17 December 2017

Janaiah Kota

10

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17 December 2017

Vinod Malik

11

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17 December 2017

Kim Shontz

12

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17 December 2017

Christopher M Walker

13

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17 December 2017

Marco Corridore

15

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17 December 2017

John R Kean

16

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17 December 2017

Hugh D Allen

17

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17 December 2017

Chris Shilling

18

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17 December 2017

Kathleen R Melia

19

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17 December 2017

Peter Sazani

20

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17 December 2017

Jay B Saoud

21

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17 December 2017

Edward M Kaye

22

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17 December 2017

Eteplirsen Study Group

23

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17 December 2017

language of work or name

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10 September 2013

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17 December 2017

published in

Annals of Neurology

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17 December 2017

volume

74

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17 December 2017

issue

5

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17 December 2017

page(s)

637-647

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17 December 2017

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

cites work

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Quality of life, physical disability, and respiratory impairment in Duchenne muscular dystrophy

21 January 2018

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Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

21 January 2018

1 reference

Systemic administration of PRO051 in Duchenne's muscular dystrophy

21 January 2018

1 reference

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

21 January 2018

1 reference

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

21 January 2018

1 reference

Dystrophin and mutations: one gene, several proteins, multiple phenotypes

21 January 2018

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Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle

21 January 2018

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Dystrophin immunity in Duchenne's muscular dystrophy.

21 January 2018

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Immunohistological intensity measurements as a tool to assess sarcolemma-associated protein expression

21 January 2018

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The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy.

21 January 2018

1 reference

Functional changes in Duchenne muscular dystrophy: a 12-month longitudinal cohort study.

21 January 2018

1 reference

The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations

21 January 2018

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Therapeutics in duchenne muscular dystrophy

21 January 2018

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A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

21 January 2018

1 reference

A randomized study of alglucosidase alfa in late-onset Pompe's disease

21 January 2018

1 reference

Six-minute-walk test in idiopathic pulmonary fibrosis: test validation and minimal clinically important difference.

21 January 2018

1 reference

Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for duchenne muscular dystrophy

21 January 2018

1 reference

Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through

21 January 2018

1 reference

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

21 January 2018

1 reference

Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.1002/ANA.23982

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17 December 2017

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17 December 2017