Literature DB >> 20880708

Choosing the right clinical outcome measure: from the patient to the statistician and back.

Eugenio Mercuri1, Elena Mazzone.   

Abstract

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Year:  2010        PMID： 20880708     DOI： 10.1016/j.nmd.2010.09.003

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


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  5 in total

1.  Reliability of the Modified Hammersmith Functional Motor Scale in young children with spinal muscular atrophy.

Authors:  Kristin J Krosschell; Charles B Scott; Jo Anne Maczulski; Aga J Lewelt; Sandra P Reyna; Kathryn J Swoboda
Journal:  Muscle Nerve       Date:  2011-06-22       Impact factor: 3.217

2.  Muscle Quantitative MR Imaging and Clustering Analysis in Patients with Facioscapulohumeral Muscular Dystrophy Type 1.

Authors:  Emilie Lareau-Trudel; Arnaud Le Troter; Badih Ghattas; Jean Pouget; Shahram Attarian; David Bendahan; Emmanuelle Salort-Campana
Journal:  PLoS One       Date:  2015-07-16       Impact factor: 3.240

Review 3.  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

4.  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

5.  A Canadian Adult Spinal Muscular Atrophy Outcome Measures Toolkit: Results of a National Consensus using a Modified Delphi Method.

Authors:  Jeremy Slayter; Victoria Hodgkinson; Josh Lounsberry; Bernard Brais; Kristine Chapman; Angela Genge; Aaron Izenberg; Wendy Johnston; Hanns Lochmüller; Erin O'Ferrall; Gerald Pfeffer; Stephanie Plamondon; Xavier Rodrigue; Kerri Schellenberg; Christen Shoesmith; Christine Stables; Monique Taillon; Jodi Warman-Chardon; Lawrence Korngut; Colleen O'Connell
Journal:  J Neuromuscul Dis       Date:  2021
  5 in total

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