Literature DB >> 25529833

204th ENMC International Workshop on Biomarkers in Duchenne Muscular Dystrophy 24-26 January 2014, Naarden, The Netherlands.

Alessandra Ferlini1, Kevin M Flanigan2, Hanns Lochmuller3, Francesco Muntoni4, Peter A C 't Hoen5, Elizabeth McNally6.   

Abstract

Entities:  

Keywords:  Biomarker; Duchenne; ENMC; Workshop

Mesh：

Substances：

Year:  2014        PMID： 25529833      PMCID： PMC4534085          DOI： 10.1016/j.nmd.2014.09.004

Source DB:  PubMed          Journal:  Neuromuscul Disord        ISSN： 0960-8966            Impact factor:   4.296


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  50 in total

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Review 2.  Voluntary exploratory data submissions to the US FDA and the EMA: experience and impact.

Authors:  Federico M Goodsaid; Shashi Amur; Jiri Aubrecht; Michael E Burczynski; Kevin Carl; Jennifer Catalano; Rosane Charlab; Sandra Close; Catherine Cornu-Artis; Laurent Essioux; Albert J Fornace; Lois Hinman; Huixiao Hong; Ian Hunt; David Jacobson-Kram; Ansar Jawaid; David Laurie; Lawrence Lesko; Heng-Hong Li; Klaus Lindpaintner; James Mayne; Peter Morrow; Marisa Papaluca-Amati; Timothy W Robison; John Roth; Ina Schuppe-Koistinen; Leming Shi; Olivia Spleiss; Weida Tong; Sharada L Truter; Jacky Vonderscher; Agnes Westelinck; Li Zhang; Issam Zineh
Journal:  Nat Rev Drug Discov       Date:  2010-06       Impact factor: 84.694

3.  The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009.

Authors:  F Muntoni
Journal:  Neuromuscul Disord       Date:  2010-03-26       Impact factor: 4.296

Review 4.  Application of genomics, proteomics and metabolomics in drug discovery, development and clinic.

Authors:  Craig Russell; Ayesha Rahman; Afzal R Mohammed
Journal:  Ther Deliv       Date:  2013-03

5.  Importance of SPP1 genotype as a covariate in clinical trials in Duchenne muscular dystrophy.

Authors:  Luca Bello; Luisa Piva; Andrea Barp; Antonella Taglia; Esther Picillo; Gessica Vasco; Marika Pane; Stefano C Previtali; Yvan Torrente; Elisabetta Gazzerro; Maria Chiara Motta; Gaetano S Grieco; Sara Napolitano; Francesca Magri; Adele D'Amico; Guja Astrea; Sonia Messina; Maria Sframeli; Gian Luca Vita; Patrizia Boffi; Tiziana Mongini; Alessandra Ferlini; Francesca Gualandi; Gianni Soraru'; Mario Ermani; Giuseppe Vita; Roberta Battini; Enrico Bertini; Giacomo P Comi; Angela Berardinelli; Carlo Minetti; Claudio Bruno; Eugenio Mercuri; Luisa Politano; Corrado Angelini; Eric P Hoffman; Elena Pegoraro
Journal:  Neurology       Date:  2012-06-27       Impact factor: 9.910

6.  DMD transcript imbalance determines dystrophin levels.

Authors:  Pietro Spitali; Janneke C van den Bergen; Ingrid E C Verhaart; Beatrijs Wokke; Anneke A M Janson; Rani van den Eijnde; Johan T den Dunnen; Jeroen F J Laros; Jan J G M Verschuuren; Peter A C 't Hoen; Annemieke Aartsma-Rus
Journal:  FASEB J       Date:  2013-08-23       Impact factor: 5.191

7.  Affinity proteomics within rare diseases: a BIO-NMD study for blood biomarkers of muscular dystrophies.

Authors:  Burcu Ayoglu; Amina Chaouch; Hanns Lochmüller; Luisa Politano; Enrico Bertini; Pietro Spitali; Monika Hiller; Eric H Niks; Francesca Gualandi; Fredrik Pontén; Kate Bushby; Annemieke Aartsma-Rus; Elena Schwartz; Yannick Le Priol; Volker Straub; Mathias Uhlén; Sebahattin Cirak; Peter A C 't Hoen; Francesco Muntoni; Alessandra Ferlini; Jochen M Schwenk; Peter Nilsson; Cristina Al-Khalili Szigyarto
Journal:  EMBO Mol Med       Date:  2014-07       Impact factor: 12.137

8.  Early neurodevelopmental assessment in Duchenne muscular dystrophy.

Authors:  Marika Pane; Roberta Scalise; Angela Berardinelli; Grazia D'Angelo; Valeria Ricotti; Paolo Alfieri; Isabella Moroni; Louise Hartley; Maria Carmela Pera; Giovanni Baranello; Michela Catteruccia; Tiziana Casalino; Domenico M Romeo; Alessandra Graziano; Claudia Gandioli; Flaviana Bianco; Elena Stacy Mazzone; Maria Elena Lombardo; Mariacristina Scoto; Serena Sivo; Concetta Palermo; Francesca Gualandi; Maria Pia Sormani; Alessandra Ferlini; Enrico Bertini; Francesco Muntoni; Eugenio Mercuri
Journal:  Neuromuscul Disord       Date:  2013-03-25       Impact factor: 4.296

9.  Matrix metalloproteinase-9 inhibition improves proliferation and engraftment of myogenic cells in dystrophic muscle of mdx mice.

Authors:  Sajedah M Hindi; Jonghyun Shin; Yuji Ogura; Hong Li; Ashok Kumar
Journal:  PLoS One       Date:  2013-08-15       Impact factor: 3.240

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

Authors:  Craig M McDonald; Erik K Henricson; R Ted Abresch; Julaine M Florence; Michelle Eagle; Eduard Gappmaier; Allan M Glanzman; Robert Spiegel; Jay Barth; Gary Elfring; Allen Reha; Stuart Peltz
Journal:  Muscle Nerve       Date:  2013-06-26       Impact factor: 3.217
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  11 in total

1.  226th ENMC International Workshop:: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20-22 January 2017, Heemskerk, The Netherlands.

Authors:  Annemieke Aartsma-Rus; Alessandra Ferlini; Elizabeth M McNally; Pietro Spitali; H Lee Sweeney
Journal:  Neuromuscul Disord       Date:  2017-10-26       Impact factor: 4.296

2.  N-terminal α Dystroglycan (αDG-N): A Potential Serum Biomarker for Duchenne Muscular Dystrophy.

Authors:  Kelly E Crowe; Guohong Shao; Kevin M Flanigan; Paul T Martin
Journal:  J Neuromuscul Dis       Date:  2016-05-27

Review 3.  Clinical utility of serum biomarkers in Duchenne muscular dystrophy.

Authors:  Yetrib Hathout; Haeri Seol; Meng Hsuan J Han; Aiping Zhang; Kristy J Brown; Eric P Hoffman
Journal:  Clin Proteomics       Date:  2016-04-05       Impact factor: 3.988

4.  Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies.

Authors:  David Israeli; Jérôme Poupiot; Fatima Amor; Karine Charton; William Lostal; Laurence Jeanson-Leh; Isabelle Richard
Journal:  Sci Rep       Date:  2016-06-21       Impact factor: 4.379

5.  Identification of serum protein biomarkers for utrophin based DMD therapy.

Authors:  Simon Guiraud; Benjamin Edwards; Sarah E Squire; Arran Babbs; Nandini Shah; Adam Berg; Huijia Chen; Kay E Davies
Journal:  Sci Rep       Date:  2017-03-02       Impact factor: 4.379

Review 6.  Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues.

Authors:  Annemieke Aartsma-Rus; Volker Straub; Robert Hemmings; Manuel Haas; Gabriele Schlosser-Weber; Violeta Stoyanova-Beninska; Eugenio Mercuri; Francesco Muntoni; Bruno Sepodes; Elizabeth Vroom; Pavel Balabanov
Journal:  Nucleic Acid Ther       Date:  2017-08-10       Impact factor: 5.486

7.  Tumor Necrosis Factor Receptor SF10A (TNFRSF10A) SNPs Correlate With Corticosteroid Response in Duchenne Muscular Dystrophy.

Authors:  Chiara Passarelli; Rita Selvatici; Alberto Carrieri; Francesca Romana Di Raimo; Maria Sofia Falzarano; Fernanda Fortunato; Rachele Rossi; Volker Straub; Katie Bushby; Mojgan Reza; Irina Zharaieva; Adele D'Amico; Enrico Bertini; Luciano Merlini; Patrizia Sabatelli; Paola Borgiani; Giuseppe Novelli; Sonia Messina; Marika Pane; Eugenio Mercuri; Mireille Claustres; Sylvie Tuffery-Giraud; Annemieke Aartsma-Rus; Pietro Spitali; Peter A C T'Hoen; Hanns Lochmüller; Kristin Strandberg; Cristina Al-Khalili; Ekaterina Kotelnikova; Michael Lebowitz; Elena Schwartz; Francesco Muntoni; Chiara Scapoli; Alessandra Ferlini
Journal:  Front Genet       Date:  2020-07-03       Impact factor: 4.599

8.  Tracking disease progression non-invasively in Duchenne and Becker muscular dystrophies.

Authors:  Pietro Spitali; Kristina Hettne; Roula Tsonaka; Mohammed Charrout; Janneke van den Bergen; Zaïda Koeks; Hermien E Kan; Melissa T Hooijmans; Andreas Roos; Volker Straub; Francesco Muntoni; Cristina Al-Khalili-Szigyarto; Marleen J A Koel-Simmelink; Charlotte E Teunissen; Hanns Lochmüller; Erik H Niks; Annemieke Aartsma-Rus
Journal:  J Cachexia Sarcopenia Muscle       Date:  2018-04-16       Impact factor: 12.910

9.  The Action Duchenne 13th International Annual Conference, November 6-7, 2015, London, UK.

Authors:  Diana Ribeiro; Daniel W Stuckey
Journal:  EBioMedicine       Date:  2015-12-03       Impact factor: 8.143

10.  Longitudinal serum biomarker screening identifies malate dehydrogenase 2 as candidate prognostic biomarker for Duchenne muscular dystrophy.

Authors:  Mirko Signorelli; Burcu Ayoglu; Camilla Johansson; Hanns Lochmüller; Volker Straub; Francesco Muntoni; Erik Niks; Roula Tsonaka; Anja Persson; Annemieke Aartsma-Rus; Peter Nilsson; Cristina Al-Khalili Szigyarto; Pietro Spitali
Journal:  J Cachexia Sarcopenia Muscle       Date:  2019-12-27       Impact factor: 12.910
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