Literature DB >> 19772190

Collaborating to bring new therapies to the patient--the TREAT-NMD model.

K Bushby1, S Lynn, T Straub.   

Abstract

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Year:  2009        PMID： 19772190      PMCID： PMC2859629     

Source DB:  PubMed          Journal:  Acta Myol        ISSN： 1128-2460


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  4 in total

1.  Cooperation is key, say neuromuscular-disease researchers.

Authors:  James Butcher
Journal:  Lancet Neurol       Date:  2007-04       Impact factor: 44.182

Review 2.  Mammalian animal models for Duchenne muscular dystrophy.

Authors:  Raffaella Willmann; Stefanie Possekel; Judith Dubach-Powell; Thomas Meier; Markus A Ruegg
Journal:  Neuromuscul Disord       Date:  2009-02-12       Impact factor: 4.296

3.  157th ENMC International Workshop: patient registries for rare, inherited muscular disorders 25-27 January 2008 Naarden, The Netherlands.

Authors:  Anna Sárközy; Kate Bushby; Christophe Béroud; Hanns Lochmüller
Journal:  Neuromuscul Disord       Date:  2008-10-22       Impact factor: 4.296

4.  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 experimental trials for DMD, 30th June--1st July 2007, Naarden, The Netherlands; conjoint Institute of Myology TREAT-NMD meeting on physical activity monitoring in neuromuscular disorders, 11th July 2007, Paris, France.

Authors:  E Mercuri; A Mayhew; F Muntoni; S Messina; V Straub; G J Van Ommen; T Voit; E Bertini; K Bushby
Journal:  Neuromuscul Disord       Date:  2008-09-24       Impact factor: 4.296
  4 in total
  18 in total

1.  Dystrophin isoform induction in vivo by antisense-mediated alternative splicing.

Authors:  Sue Fletcher; Abbie M Adams; Russell D Johnsen; Kane Greer; Hong M Moulton; Steve D Wilton
Journal:  Mol Ther       Date:  2010-03-23       Impact factor: 11.454

2.  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

Review 3.  Can outcomes in Duchenne muscular dystrophy be improved by public reporting of data?

Authors:  Michele A Scully; Valerie A Cwik; Bruce C Marshall; Emma Ciafaloni; Jodi M Wolff; Thomas S Getchius; Robert C Griggs
Journal:  Neurology       Date:  2013-02-05       Impact factor: 9.910

Review 4.  Limb-girdle muscular dystrophies - international collaborations for translational research.

Authors:  Rachel Thompson; Volker Straub
Journal:  Nat Rev Neurol       Date:  2016-04-01       Impact factor: 42.937

Review 5.  RD-Connect: an integrated platform connecting databases, registries, biobanks and clinical bioinformatics for rare disease research.

Authors:  Rachel Thompson; Louise Johnston; Domenica Taruscio; Lucia Monaco; Christophe Béroud; Ivo G Gut; Mats G Hansson; Peter-Bram A 't Hoen; George P Patrinos; Hugh Dawkins; Monica Ensini; Kurt Zatloukal; David Koubi; Emma Heslop; Justin E Paschall; Manuel Posada; Peter N Robinson; Kate Bushby; Hanns Lochmüller
Journal:  J Gen Intern Med       Date:  2014-08       Impact factor: 5.128

Review 6.  The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review.

Authors:  Christina Q Nguyen; Kristine Alba-Concepcion; Elizabeth E Palmer; Jackie L Scully; Nicole Millis; Michelle A Farrar
Journal:  Orphanet J Rare Dis       Date:  2022-04-18       Impact factor: 4.303

7.  Early stages of building a rare disease registry, methods and 2010 data from the Belgian Neuromuscular Disease Registry (BNMDR).

Authors:  Anna J Roy; Peter Van den Bergh; Philip Van Damme; Kris Doggen; Viviane Van Casteren
Journal:  Acta Neurol Belg       Date:  2014-06-24       Impact factor: 2.396

8.  The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations.

Authors:  Catherine L Bladen; David Salgado; Soledad Monges; Maria E Foncuberta; Kyriaki Kekou; Konstantina Kosma; Hugh Dawkins; Leanne Lamont; Anna J Roy; Teodora Chamova; Velina Guergueltcheva; Sophelia Chan; Lawrence Korngut; Craig Campbell; Yi Dai; Jen Wang; Nina Barišić; Petr Brabec; Jaana Lahdetie; Maggie C Walter; Olivia Schreiber-Katz; Veronika Karcagi; Marta Garami; Venkatarman Viswanathan; Farhad Bayat; Filippo Buccella; En Kimura; Zaïda Koeks; Janneke C van den Bergen; Miriam Rodrigues; Richard Roxburgh; Anna Lusakowska; Anna Kostera-Pruszczyk; Janusz Zimowski; Rosário Santos; Elena Neagu; Svetlana Artemieva; Vedrana Milic Rasic; Dina Vojinovic; Manuel Posada; Clemens Bloetzer; Pierre-Yves Jeannet; Franziska Joncourt; Jordi Díaz-Manera; Eduard Gallardo; A Ayşe Karaduman; Haluk Topaloğlu; Rasha El Sherif; Angela Stringer; Andriy V Shatillo; Ann S Martin; Holly L Peay; Matthew I Bellgard; Jan Kirschner; Kevin M Flanigan; Volker Straub; Kate Bushby; Jan Verschuuren; Annemieke Aartsma-Rus; Christophe Béroud; Hanns Lochmüller
Journal:  Hum Mutat       Date:  2015-03-17       Impact factor: 4.878

9.  Design, set-up and utility of the UK facioscapulohumeral muscular dystrophy patient registry.

Authors:  Teresinha Evangelista; Libby Wood; Roberto Fernandez-Torron; Maggie Williams; Debbie Smith; Peter Lunt; Judith Hudson; Fiona Norwood; Richard Orrell; Tracey Willis; David Hilton-Jones; Karen Rafferty; Michela Guglieri; Hanns Lochmüller
Journal:  J Neurol       Date:  2016-05-09       Impact factor: 4.849

10.  The Canadian Neuromuscular Disease Registry: Connecting patients to national and international research opportunities.

Authors:  Yi Wei; Anna McCormick; Alex MacKenzie; Erin O'Ferrall; Shannon Venance; Jean K Mah; Kathryn Selby; Hugh J McMillan; Garth Smith; Maryam Oskoui; Gillian Hogan; Laura McAdam; Gracia Mabaya; Victoria Hodgkinson; Josh Lounsberry; Lawrence Korngut; Craig Campbell
Journal:  Paediatr Child Health       Date:  2017-12-08       Impact factor: 2.253
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