Journal article
227th ENMC International Workshop:: Finalizing a plan to guarantee quality in translational research for neuromuscular diseases Heemskerk, Netherlands, 10–11 February 2017
Neuromuscular disorders : NMD, Vol.28(2), pp.185-192
02/2018
DOI: 10.1016/j.nmd.2017.11.002
PMID: 29361397
Abstract
•The issue of translatability of pre-clinical results was discussed with a wide range of stakeholders.•Challenges and pitfalls regarding the planning, funding, reporting and publishing of efficacy studies were discussed.•Future common efforts were planned to improve translatability of pre-clinical research for neuromuscular disorders.
Details
- Title: Subtitle
- 227th ENMC International Workshop:: Finalizing a plan to guarantee quality in translational research for neuromuscular diseases Heemskerk, Netherlands, 10–11 February 2017
- Creators
- Victor Dubowitz - Neuromuscular Disorders, UKHanns Lochmüller - Journal of Neuromuscular Diseases, UKKevin Campbell - Skeletal Muscle, USAKay Davies - Human Molecular Genetics, UKKevin A Roth - American Journal of Pathology, USAAlejandra Clark - PLOS ONE, USAEmilio Clementi - Pharmacological Research, ItalyKanneboyina Nagaraju - School of Pharmacy and Pharmaceutical Sciences, Binghamton University, New York, USANathalie Goemans - University Hospital Leuven, BelgiumVolker Straub - University of Newcastle, UKRaffaella Willmann - University of Basel and Swiss Foundation for Research on Muscle Diseases, SwitzerlandFilippo Buccella - Duchenne Parent Project, ItalyAndrea Klein - Universität-Kinderspital Beider Basel, Inselspital Bern and CHUV Lausanne, SwitzerlandAnnamaria De Luca - University of Bari, Bari, ItalyAnnemieke Aartsma-Rus - Leiden University Medical Center, NetherlandsMiranda Grounds - University of Western Australia, AustraliaMaaike van Putten - Leiden University Medical Center, NetherlandsJenny Versnel - Muscular Dystrophy, UKElizabeth Vroom - United Parent Project Muscular Dystrophy, NetherlandsMaria Fries - Progena Foundation, SwitzerlandDiana Ribeiro - Action Duchenne, UKMaria Sheean - European Medicines Agency (EMA), London, UKAnna Ambrosini - Fondazione Telethon, ItalyJon Tinsley - Summit Therapeutics, UKGrace Pavlath - Muscular Dystrophy Association, USAMahasweta Girgenrath - Pfizer, USAJohn Porter - Myotonic Dystrophy Foundation, USAGustavo Dziewczapolski - CureCMD, USA
- Resource Type
- Journal article
- Publication Details
- Neuromuscular disorders : NMD, Vol.28(2), pp.185-192
- DOI
- 10.1016/j.nmd.2017.11.002
- PMID
- 29361397
- NLM abbreviation
- Neuromuscul Disord
- ISSN
- 0960-8966
- eISSN
- 1873-2364
- Publisher
- Elsevier B.V
- Grant note
- DOI: 10.13039/501100011640, name: European Neuromuscular Centre (ENMC); name: Association Française contre les Myopathies (France); DOI: 10.13039/501100010275, name: Deutsche Gesellschaft für Muskelkranke (Germany); name: Muscular Dystrophy UK (UK); name: Muskelsvindfonden (Denmark); name: Prinses Beatrix Spierfonds (Netherlands); name: Schweizerische Stiftung für die Erforschung der Muskelkrankheiten (Switzerland); name: Telethon Foundation (Italy); name: Spierziekten Nederland (Netherlands); name: Finnish Neuromuscular Association (Finland); name: Parent Project Muscular Dystrophy USA
- Language
- English
- Date published
- 02/2018
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Iowa Neuroscience Institute
- Record Identifier
- 9984068375002771
Metrics
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