Literature DB >> 17433676

145th ENMC International Workshop: planning for an International Trial of Steroid Dosage Regimes in DMD (FOR DMD), 22-24th October 2006, Naarden, The Netherlands.

K Bushby1, R Griggs.   

Abstract

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Year:  2007        PMID： 17433676     DOI： 10.1016/j.nmd.2007.01.006

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


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  7 in total

1.  The Canadian experience with long-term deflazacort treatment in Duchenne muscular dystrophy.

Authors:  Laura C McAdam; Amanda L Mayo; Benjamin A Alman; W Douglas Biggar
Journal:  Acta Myol       Date:  2012-05

2.  Myostatin inhibition by a follistatin-derived peptide ameliorates the pathophysiology of muscular dystrophy model mice.

Authors:  K Tsuchida
Journal:  Acta Myol       Date:  2008-07

Review 3.  Therapeutic possibilities in the autosomal recessive limb-girdle muscular dystrophies.

Authors:  Volker Straub; Kate Bushby
Journal:  Neurotherapeutics       Date:  2008-10       Impact factor: 7.620

Review 4.  The Limb-Girdle Muscular Dystrophies: Is Treatment on the Horizon?

Authors:  Mary Lynn Chu; Ellen Moran
Journal:  Neurotherapeutics       Date:  2018-10       Impact factor: 7.620

Review 5.  A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial.

Authors:  Rebecca A Crow; Kimberly A Hart; Michael P McDermott; Rabi Tawil; William B Martens; Barbara E Herr; Elaine McColl; Jennifer Wilkinson; Janbernd Kirschner; Wendy M King; Michele Eagle; Mary W Brown; Deborah Hirtz; Hanns Lochmuller; Volker Straub; Emma Ciafaloni; Perry B Shieh; Stefan Spinty; Anne-Marie Childs; Adnan Y Manzur; Lucia Morandi; Russell J Butterfield; Iain Horrocks; Helen Roper; Kevin M Flanigan; Nancy L Kuntz; Jean K Mah; Leslie Morrison; Basil T Darras; Maja von der Hagen; Ulrike Schara; Ekkehard Wilichowski; Tiziana Mongini; Craig M McDonald; Giuseppe Vita; Richard J Barohn; Richard S Finkel; Matthew Wicklund; Hugh J McMillan; Imelda Hughes; Elena Pegoraro; W Bryan Burnette; James F Howard; Mathula Thangarajh; Craig Campbell; Robert C Griggs; Kate Bushby; Michela Guglieri
Journal:  Trials       Date:  2018-05-10       Impact factor: 2.279

6.  Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents.

Authors:  Steve D Wilton; Sue Fletcher
Journal:  Appl Clin Genet       Date:  2011-03-10

7.  Treatment of dysferlinopathy with deflazacort: a double-blind, placebo-controlled clinical trial.

Authors:  Maggie C Walter; Peter Reilich; Simone Thiele; Joachim Schessl; Herbert Schreiber; Karlheinz Reiners; Wolfram Kress; Clemens Müller-Reible; Matthias Vorgerd; Peter Urban; Bertold Schrank; Marcus Deschauer; Beate Schlotter-Weigel; Ralf Kohnen; Hanns Lochmüller
Journal:  Orphanet J Rare Dis       Date:  2013-02-14       Impact factor: 4.123
  7 in total

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