Literature DB >> 25307856

Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy.

Stephen Lynn1, Annemieke Aartsma-Rus2, Kate Bushby1, Pat Furlong3, Nathalie Goemans4, Annamaria De Luca5, Anna Mayhew1, Craig McDonald6, Eugenio Mercuri7, Francesco Muntoni8, Marita Pohlschmidt9, Jan Verschuuren10, Thomas Voit11, Elizabeth Vroom12, Dominic J Wells13, Volker Straub14.   

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Year:  2014        PMID: 25307856     DOI: 10.1016/j.nmd.2014.09.003

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


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  17 in total

1.  Increasing taurine intake and taurine synthesis improves skeletal muscle function in the mdx mouse model for Duchenne muscular dystrophy.

Authors:  Jessica R Terrill; Gavin J Pinniger; Jamie A Graves; Miranda D Grounds; Peter G Arthur
Journal:  J Physiol       Date:  2016-01-18       Impact factor: 5.182

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.  Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials.

Authors:  Pierre G Carlier; Benjamin Marty; Olivier Scheidegger; Paulo Loureiro de Sousa; Pierre-Yves Baudin; Eduard Snezhko; Dmitry Vlodavets
Journal:  J Neuromuscul Dis       Date:  2016-03-03

4.  Magnetic resonance imaging of the proximal upper extremity musculature in boys with Duchenne muscular dystrophy.

Authors:  R J Willcocks; W T Triplett; S C Forbes; H Arora; C R Senesac; D J Lott; T R Nicholson; W D Rooney; G A Walter; K Vandenborne
Journal:  J Neurol       Date:  2016-10-24       Impact factor: 4.849

5.  Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.

Authors:  Rebecca J Willcocks; William D Rooney; William T Triplett; Sean C Forbes; Donovan J Lott; Claudia R Senesac; Michael J Daniels; Dah-Jyuu Wang; Ann T Harrington; Gihan I Tennekoon; Barry S Russman; Erika L Finanger; Barry J Byrne; Richard S Finkel; Glenn A Walter; H Lee Sweeney; Krista Vandenborne
Journal:  Ann Neurol       Date:  2016-02-19       Impact factor: 10.422

6.  Longitudinal Alterations in Gait Features in Growing Children With Duchenne Muscular Dystrophy.

Authors:  Ines Vandekerckhove; Marleen Van den Hauwe; Nathalie De Beukelaer; Elze Stoop; Marije Goudriaan; Margaux Delporte; Geert Molenberghs; Anja Van Campenhout; Liesbeth De Waele; Nathalie Goemans; Friedl De Groote; Kaat Desloovere
Journal:  Front Hum Neurosci       Date:  2022-06-02       Impact factor: 3.473

7.  Improving clinical trial design for Duchenne muscular dystrophy.

Authors:  Luciano Merlini; Patrizia Sabatelli
Journal:  BMC Neurol       Date:  2015-08-26       Impact factor: 2.474

8.  Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test.

Authors:  Marika Pane; Lavinia Fanelli; Elena Stacy Mazzone; Giorgia Olivieri; Adele D'Amico; Sonia Messina; Marianna Scutifero; Roberta Battini; Roberta Petillo; Silvia Frosini; Serena Sivo; Gian Luca Vita; Claudio Bruno; Tiziana Mongini; Elena Pegoraro; Roberto De Sanctis; Alice Gardani; Angela Berardinelli; Valentina Lanzillotta; Adelina Carlesi; Emanuela Viggiano; Filippo Cavallaro; Maria Sframeli; Luca Bello; Andrea Barp; Flaviana Bianco; Serena Bonfiglio; Enrica Rolle; Concetta Palermo; Grazia D'Angelo; Antonella Pini; Elena Iotti; Ksenija Gorni; Giovanni Baranello; Enrico Bertini; Luisa Politano; Maria Pia Sormani; Eugenio Mercuri
Journal:  Neuromuscul Disord       Date:  2015-07-17       Impact factor: 4.296

9.  Muscle-Derived Proteins as Serum Biomarkers for Monitoring Disease Progression in Three Forms of Muscular Dystrophy.

Authors:  Peter M Burch; Oksana Pogoryelova; Richard Goldstein; Donald Bennett; Michela Guglieri; Volker Straub; Kate Bushby; Hanns Lochmüller; Carl Morris
Journal:  J Neuromuscul Dis       Date:  2015-09-02

10.  Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials.

Authors:  Eugenio Mercuri; Richard Finkel; Jacqueline Montes; Elena S Mazzone; Maria Pia Sormani; Marion Main; Danielle Ramsey; Anna Mayhew; Allan M Glanzman; Sally Dunaway; Rachel Salazar; Amy Pasternak; Janet Quigley; Marika Pane; Maria Carmela Pera; Mariacristina Scoto; Sonia Messina; Maria Sframeli; Gian Luca Vita; Adele D'Amico; Marleen van den Hauwe; Serena Sivo; Nathalie Goemans; Petra Kaufmann; Basil T Darras; Enrico Bertini; Francesco Muntoni; Darryl C De Vivo
Journal:  Neuromuscul Disord       Date:  2015-12-03       Impact factor: 4.296

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