The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia (Q38406108)

2 September 2017

title

The TREAT-NMD Duchenne muscular dystrophy registries: conception, design, and utilization by industry and academia (English)

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Duchenne muscular dystrophy

2 September 2017

main subject

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60

Domenica Taruscio

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Ann Martin

Ann S Martin

52

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Richard H. Roxburgh

Richard Roxburgh

34

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Sabina Gainotti

Sabina Gainotti

59

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Rosário Santos

Rosário Santos

37

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Jorge Oliveira

Jorge Oliveira

36

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Rabah Ben Yaou

Rabah Ben Yaou

21

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En Kimura

En Kimura

31

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Kevin M. Flanigan

Kevin M Flanigan

54

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Christophe Beroud

Christophe Béroud

64

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Janbernd Kirschner

Jan Kirschner

61

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Francesc Palau

Francesc Palau

43

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Manuel Posada

Manuel Posada

42

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Hanns Lochmüller

Hanns Lochmüller

65

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David Salgado

David Salgado

55

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Volker Straub

3

Volker Straub

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2 September 2017

19

Mireille Claustres

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2 September 2017

Sylvie Tuffery-Giraud

18

Sylvie Tuffery-Giraud

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2 September 2017

35

Anna Lusakowska

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2 September 2017

Catherine L Bladen

1

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2 September 2017

Karen Rafferty

2

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2 September 2017

Soledad Monges

4

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2 September 2017

Angélica Moresco

5

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2 September 2017

Hugh Dawkins

6

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2 September 2017

Anna Roy

7

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2 September 2017

Teodora Chamova

8

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2 September 2017

Velina Guergueltcheva

9

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2 September 2017

Lawrence Korngut

10

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2 September 2017

Craig Campbell

11

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2 September 2017

Yi Dai

12

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2 September 2017

Nina Barišić

13

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2 September 2017

Tea Kos

14

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2 September 2017

Petr Brabec

15

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2 September 2017

Jes Rahbek

16

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2 September 2017

Jaana Lahdetie

17

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2 September 2017

France Leturcq

20

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2 September 2017

Maggie C Walter

22

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2 September 2017

Olivia Schreiber

23

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2 September 2017

Veronika Karcagi

24

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2 September 2017

Agnes Herczegfalvi

25

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2 September 2017

Venkatarman Viswanathan

26

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2 September 2017

Farhad Bayat

27

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2 September 2017

Isis de la Caridad Guerrero Sarmiento

28

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2 September 2017

Anna Ambrosini

29

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2 September 2017

Francesca Ceradini

30

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2 September 2017

Janneke C van den Bergen

32

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2 September 2017

Miriam Rodrigues

33

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2 September 2017

Elena Neagu

38

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2 September 2017

Niculina Butoianu

39

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2 September 2017

Svetlana Artemieva

40

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2 September 2017

Vedrana Milic Rasic

41

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2 September 2017

Björn Lindvall

44

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2 September 2017

Clemens Bloetzer

45

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2 September 2017

Ayşe Karaduman

46

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2 September 2017

Haluk Topaloğlu

47

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2 September 2017

Serap Inal

48

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2 September 2017

Piraye Oflazer

49

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2 September 2017

Angela Stringer

50

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2 September 2017

Andriy V Shatillo

51

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2 September 2017

Holly Peay

53

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2 September 2017

Brigitta von Rekowski

56

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2 September 2017

Stephen Lynn

57

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2 September 2017

Emma Heslop

58

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2 September 2017

Jan Verschuuren

62

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2 September 2017

Kate Bushby

63

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2 September 2017

publication date

26 August 2013

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2 September 2017

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2 September 2017

volume

34

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2 September 2017

issue

11

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2 September 2017

page(s)

1449-1457

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

2 September 2017

cites work

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

The structural and functional diversity of dystrophin

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Characterization of the DMD/BMD patient population in Czech Republic and Slovakia using an innovative registry approach

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Mutation analysis in a population-based cohort of boys with Duchenne or Becker muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Duchenne muscular dystrophy: a 30-year population-based incidence study.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Duchenne muscular dystrophy--Meryon's disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Corticosteroids in Duchenne muscular dystrophy: major variations in practice

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy).

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

157th ENMC International Workshop: patient registries for rare, inherited muscular disorders 25-27 January 2008 Naarden, The Netherlands

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Can outcomes in Duchenne muscular dystrophy be improved by public reporting of data?

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Standards of care for Duchenne muscular dystrophy: brief TREAT-NMD recommendations

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Genotype-phenotype analysis in 2,405 patients with a dystrophinopathy using the UMD-DMD database: a model of nationwide knowledgebase

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Advances in therapeutic RNA-targeting

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Copy number variation in the genome; the human DMD gene as an example

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

Treatment of the heart in Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

7 January 2021

Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

7 January 2021

The muscular dystrophies

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

7 January 2021

Towards harmonization of protocols for MRI outcome measures in skeletal muscle studies: consensus recommendations from two TREAT-NMD NMR workshops, 2 May 2010, Stockholm, Sweden, 1-2 October 2009, Paris, France

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

7 January 2021

Motor and respiratory heterogeneity in Duchenne patients: Implication for clinical trials

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

7 January 2021

The New Zealand Neuromuscular Disease Registry

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

7 January 2021

TREAT-NMD workshop: pattern recognition in genetic muscle diseases using muscle MRI: 25-26 February 2011, Rome, Italy

1 reference

https://api.crossref.org/works/10.1002%2FHUMU.22390

7 January 2021

Identifiers

DOI

10.1002/HUMU.22390

1 reference

release_ykhkunvv7bdlfhgqltie5ew2qa

2 September 2017

Fatcat ID

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Fatcat

https://api.fatcat.wiki/v0/release/ykhkunvv7bdlfhgqltie5ew2qa

24 November 2022

mapped directly with Wikidata item

1 reference