(Q45033314)

English

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

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scholarly article

1 reference

Europe PubMed Central

PubMed publication ID

8 December 2017

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study (English)

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Europe PubMed Central

PubMed publication ID

8 December 2017

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inferred from title

Duchenne muscular dystrophy

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3

object named as

Volker Straub

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8 December 2017

Haluk Topaloglu

2

object named as

Haluk Topaloglu

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8 December 2017

author name string

Thomas Voit

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8 December 2017

Francesco Muntoni

4

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8 December 2017

Nicolas Deconinck

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8 December 2017

Giles Campion

6

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8 December 2017

Sjef J De Kimpe

7

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8 December 2017

Michelle Eagle

8

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8 December 2017

Michela Guglieri

9

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8 December 2017

Steve Hood

10

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Lia Liefaard

11

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8 December 2017

Afrodite Lourbakos

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8 December 2017

Allison Morgan

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Joanna Nakielny

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8 December 2017

Naashika Quarcoo

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8 December 2017

Valeria Ricotti

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Katie Rolfe

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8 December 2017

Laurent Servais

18

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Claire Wardell

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Rosamund Wilson

20

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Padraig Wright

21

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8 December 2017

John E Kraus

22

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8 December 2017

language of work or name

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publication date

7 September 2014

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8 December 2017

Lancet Neurology

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8 December 2017

13

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8 December 2017

10

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8 December 2017

987-996

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8 December 2017

195th ENMC International Workshop: Newborn screening for Duchenne muscular dystrophy 14-16th December, 2012, Naarden, The Netherlands

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https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Ambulatory capacity and disease progression as measured by the 6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily corticosteroids

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

6 Minute walk test in Duchenne MD patients with different mutations: 12 month changes

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Eteplirsen for the treatment of Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Systemic administration of PRO051 in Duchenne's muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

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%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

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

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Monoclonal antibodies against defined regions of the muscular dystrophy protein, dystrophin

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2814%2970195-4

7 January 2021

based on heuristic

inferred from DOI database lookup

Identifiers

10.1016/S1474-4422(14)70195-4

1 reference

Europe PubMed Central

PubMed publication ID

8 December 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

8 December 2017

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