226th ENMC International Workshop:: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20–22 January 2017, Heemskerk, The Netherlands

workshop participants

doi:10.1016/j.nmd.2017.10.002

226^th ENMC International Workshop:: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20–22 January 2017, Heemskerk, The Netherlands

workshop participants

Medicine, Cardiology Division

Research output: Contribution to journal › Article › peer-review

9 Scopus citations

Original language	English (US)
Pages (from-to)	77-86
Number of pages	10
Journal	Neuromuscular Disorders
Volume	28
Issue number	1
DOIs	https://doi.org/10.1016/j.nmd.2017.10.002
State	Published - Jan 1 2018

Keywords

Biobank
Biomarker
Duchenne muscular dystrophy
Dystrophin
MRI

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Neurology
Clinical Neurology
Genetics(clinical)

Access to Document

10.1016/j.nmd.2017.10.002

Cite this

@article{66a47185cd0541449baa66c8e24d6417,

title = "226th ENMC International Workshop:: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20–22 January 2017, Heemskerk, The Netherlands",

keywords = "Biobank, Biomarker, Duchenne muscular dystrophy, Dystrophin, MRI",

author = "{workshop participants} and Annemieke Aartsma-Rus and Alessandra Ferlini and McNally, {Elizabeth M.} and Pietro Spitali and Sweeney, {H. Lee} and Szigyarto, {Christina Al Khalili} and Luca Bello and Abby Bronson and Kristy Brown and Filippo Buccella and Jessica Chadwick and Diane Frank and Eric Hoffman and Jane Larkindale and G. McClorey and Rick Munschauer and Francesco Muntoni and Jane Owens and Ulrike Schara and Volker Straub and Jon Tinsley and Jenny Versnel and Elizabeth Vroom and Ellen Welch",

note = "Funding Information: This Workshop was made possible thanks to the financial support of the European Neuromuscular Centre (ENMC) and ENMC main sponsors: Funding Information: The workshop was organized with the support of Parent Project Muscular Dystrophy (PPMD) and Marathon Pharmaceuticals, which provided travel support for participants from the US via an unrestricted grant to PPMD in addition to ENMC support. It was attended by representatives of academic institutions, industry working in the Duchenne muscular dystrophy field and patient representatives. Funding Information: Pietro Spitali presented on an effort that was initiated after the 204th ENMC workshop to make an inventory of serum and plasma samples available for biomarker validation studies. The work was sponsored by a small grant from Duchenne Parent Project Netherlands. Samples have been identified, some of which are linked to functional data and some of which are longitudinal. It is clear that more effort is needed to collect additional samples and functional data in a controlled way. Samples collected in placebo arms of clinical trials would be ideal to validate candidate biomarkers. ",

year = "2018",

month = jan,

day = "1",

doi = "10.1016/j.nmd.2017.10.002",

language = "English (US)",

volume = "28",

pages = "77--86",

journal = "Neuromuscular Disorders",

issn = "0960-8966",

publisher = "Elsevier Limited",

number = "1",

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T1 - 226th ENMC International Workshop:: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20–22 January 2017, Heemskerk, The Netherlands

AU - workshop participants

AU - Aartsma-Rus, Annemieke

AU - Ferlini, Alessandra

AU - McNally, Elizabeth M.

AU - Spitali, Pietro

AU - Sweeney, H. Lee

AU - Szigyarto, Christina Al Khalili

AU - Bello, Luca

AU - Bronson, Abby

AU - Brown, Kristy

AU - Buccella, Filippo

AU - Chadwick, Jessica

AU - Frank, Diane

AU - Hoffman, Eric

AU - Larkindale, Jane

AU - McClorey, G.

AU - Munschauer, Rick

AU - Muntoni, Francesco

AU - Owens, Jane

AU - Schara, Ulrike

AU - Straub, Volker

AU - Tinsley, Jon

AU - Versnel, Jenny

AU - Vroom, Elizabeth

AU - Welch, Ellen

N1 - Funding Information: This Workshop was made possible thanks to the financial support of the European Neuromuscular Centre (ENMC) and ENMC main sponsors: Funding Information: The workshop was organized with the support of Parent Project Muscular Dystrophy (PPMD) and Marathon Pharmaceuticals, which provided travel support for participants from the US via an unrestricted grant to PPMD in addition to ENMC support. It was attended by representatives of academic institutions, industry working in the Duchenne muscular dystrophy field and patient representatives. Funding Information: Pietro Spitali presented on an effort that was initiated after the 204th ENMC workshop to make an inventory of serum and plasma samples available for biomarker validation studies. The work was sponsored by a small grant from Duchenne Parent Project Netherlands. Samples have been identified, some of which are linked to functional data and some of which are longitudinal. It is clear that more effort is needed to collect additional samples and functional data in a controlled way. Samples collected in placebo arms of clinical trials would be ideal to validate candidate biomarkers.

PY - 2018/1/1

Y1 - 2018/1/1

KW - Biobank

KW - Biomarker

KW - Duchenne muscular dystrophy

KW - Dystrophin

KW - MRI

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U2 - 10.1016/j.nmd.2017.10.002

DO - 10.1016/j.nmd.2017.10.002

M3 - Article

C2 - 29203356

AN - SCOPUS:85035314174

SN - 0960-8966

VL - 28

SP - 77

EP - 86

JO - Neuromuscular Disorders

JF - Neuromuscular Disorders

IS - 1