Literature DB >> 21115346

Developing standard procedures for pre-clinical efficacy studies in mouse models of spinal muscular atrophy: report of the expert workshop "Pre-clinical testing for SMA", Zürich, March 29-30th 2010.

Raffaella Willmann1, Judith Dubach, Karen Chen.   

Abstract

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Year:  2010        PMID： 21115346     DOI： 10.1016/j.nmd.2010.09.014

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


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  12 in total

1.  Chronic exercise mitigates disease mechanisms and improves muscle function in myotonic dystrophy type 1 mice.

Authors:  Alexander Manta; Derek W Stouth; Donald Xhuti; Leon Chi; Irena A Rebalka; Jayne M Kalmar; Thomas J Hawke; Vladimir Ljubicic
Journal:  J Physiol       Date:  2019-01-30       Impact factor: 5.182

2.  Transgenic expression of neuronal dystonin isoform 2 partially rescues the disease phenotype of the dystonia musculorum mouse model of hereditary sensory autonomic neuropathy VI.

Authors:  Andrew Ferrier; Tadasu Sato; Yves De Repentigny; Sabrina Gibeault; Kunal Bhanot; Ryan W O'Meara; Anisha Lynch-Godrei; Samantha F Kornfeld; Kevin G Young; Rashmi Kothary
Journal:  Hum Mol Genet       Date:  2013-12-30       Impact factor: 6.150

3.  Severe neuromuscular denervation of clinically relevant muscles in a mouse model of spinal muscular atrophy.

Authors:  Karen K Y Ling; Rebecca M Gibbs; Zhihua Feng; Chien-Ping Ko
Journal:  Hum Mol Genet       Date:  2011-10-03       Impact factor: 6.150

Review 4.  Pathophysiological concepts in the congenital myopathies: blurring the boundaries, sharpening the focus.

Authors:  Gianina Ravenscroft; Nigel G Laing; Carsten G Bönnemann
Journal:  Brain       Date:  2014-12-31       Impact factor: 13.501

5.  Fasudil improves survival and promotes skeletal muscle development in a mouse model of spinal muscular atrophy.

Authors:  Melissa Bowerman; Lyndsay M Murray; Justin G Boyer; Carrie L Anderson; Rashmi Kothary
Journal:  BMC Med       Date:  2012-03-07       Impact factor: 8.775

Review 6.  The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development.

Authors:  Emma Heslop; Cristina Csimma; Volker Straub; John McCall; Kanneboyina Nagaraju; Kathryn R Wagner; Didier Caizergues; Rudolf Korinthenberg; Kevin M Flanigan; Petra Kaufmann; Elizabeth McNeil; Jerry Mendell; Sharon Hesterlee; Dominic J Wells; Kate Bushby
Journal:  Orphanet J Rare Dis       Date:  2015-04-23       Impact factor: 4.123

7.  Looking Forward to New Therapies: A Personal Perspective on the Translational Landscape for Muscular Dystrophies.

Authors:  Kate Bushby
Journal:  J Neuromuscul Dis       Date:  2015-07-22

8.  Update on Standard Operating Procedures in Preclinical Research for DMD and SMA Report of TREAT-NMD Alliance Workshop, Schiphol Airport, 26 April 2015, The Netherlands.

Authors:  Maaike van Putten; Annemieke Aartsma-Rus; Miranda D Grounds; Joe N Kornegay; Anna Mayhew; Thomas H Gillingwater; Shin'ichi Takeda; Markus A Rüegg; Annamaria De Luca; Kanneboyina Nagaraju; Raffaella Willmann
Journal:  J Neuromuscul Dis       Date:  2018

Review 9.  Mouse models for muscular dystrophies: an overview.

Authors:  Maaike van Putten; Erin M Lloyd; Jessica C de Greef; Vered Raz; Raffaella Willmann; Miranda D Grounds
Journal:  Dis Model Mech       Date:  2020-02-21       Impact factor: 5.758

Review 10.  Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT).

Authors:  Raffaella Willmann; Joanne Lee; Cathy Turner; Kanneboyina Nagaraju; Annemieke Aartsma-Rus; Dominic J Wells; Kathryn R Wagner; Cristina Csimma; Volker Straub; Miranda D Grounds; Annamaria De Luca
Journal:  Dis Model Mech       Date:  2020-02-07       Impact factor: 5.758
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