(Q24616493)

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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

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scholarly article

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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 (English)

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Duchenne muscular dystrophy

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inferred from title

Francesco Muntoni

20

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Sebahattin Çırak

1

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Maria Elena Garralda

8

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Virginia Arechavala-Gomeza

2

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object named as

Karen Anthony

6

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Dominic J Wells

object named as

Dominic J Wells

10

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14

object named as

Volker Straub

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15

object named as

Ryszard Kole

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Michela Guglieri

3

object named as

Michela Guglieri

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4

object named as

Lucy Feng

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5

object named as

Silvia Torelli

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Caroline A Sewry

17

object named as

Caroline Sewry

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Matthew J. Wood

12

object named as

Matthew J A Wood

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author name string

Stephen Abbs

7

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John Bourke

9

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George Dickson

11

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Steve D Wilton

13

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Stephen B Shrewsbury

16

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Jennifer E Morgan

18

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Kate Bushby

19

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language of work or name

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publication date

13 August 2011

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378

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9791

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595-605

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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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

7 April 2017

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

7 April 2017

Systemic administration of PRO051 in Duchenne's muscular dystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

Muscle histology vs MRI in Duchenne muscular dystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

Exon-skipped dystrophins for treatment of Duchenne muscular dystrophy: mass spectrometry mapping of most exons and cooperative domain designs based on single molecule mechanics

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx Mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

Reliability of the North Star Ambulatory Assessment in a multicentric setting.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

Sarcolemma-localized nNOS is required to maintain activity after mild exercise

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

Dystrophin gene abnormalities in two patients with idiopathic dilated cardiomyopathy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

Use of epitope libraries to identify exon-specific monoclonal antibodies for characterization of altered dystrophins in muscular dystrophy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

27 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

28 November 2018

Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

28 November 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

28 November 2018

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

28 November 2018

Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

28 November 2018

Gene expression profiling in the early phases of DMD: a constant molecular signature characterizes DMD muscle from early postnatal life throughout disease progression

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

28 November 2018

Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

28 November 2018

Grandpa and I have dystrophinopathy?: approach to asymptomatic hyperCKemia.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

28 November 2018

Use of step activity monitoring for continuous physical activity assessment in boys with Duchenne muscular dystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3156980

28 November 2018

The clinical, genetic and dystrophin characteristics of Becker muscular dystrophy. II. Correlation of phenotype with genetic and protein abnormalities

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2811%2960756-3

7 January 2021

based on heuristic

inferred from DOI database lookup

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2811%2960756-3

7 January 2021

based on heuristic

inferred from DOI database lookup

Monoclonal antibody analysis of mononuclear cells in myopathies. I: Quantitation of subsets according to diagnosis and sites of accumulation and demonstration and counts of muscle fibers invaded by T cells

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2811%2960756-3

7 January 2021

based on heuristic

inferred from DOI database lookup

Becker muscular dystrophy patients with deletions around exon 51; a promising outlook for exon skipping therapy in Duchenne patients

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2811%2960756-3

7 January 2021

based on heuristic

inferred from DOI database lookup

Dystrophin characterization in BMD patients: correlation of abnormal protein with clinical phenotype

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2811%2960756-3

7 January 2021

based on heuristic

inferred from DOI database lookup

Safety pharmacology and genotoxicity evaluation of AVI-4658

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2811%2960756-3

7 January 2021

based on heuristic

inferred from DOI database lookup

MR imaging in Duchenne muscular dystrophy: quantification of T1-weighted signal, contrast uptake, and the effects of exercise

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2811%2960756-3

7 January 2021

based on heuristic

inferred from DOI database lookup

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