Literature DB >> 18207401

149th ENMC International Workshop and 1st TREAT-NMD Workshop on: "planning phase i/ii clinical trials using systemically delivered antisense oligonucleotides in duchenne muscular dystrophy".

Francesco Muntoni1, Kate D Bushby, Gertjan van Ommen.   

Abstract

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Year:  2008        PMID： 18207401     DOI： 10.1016/j.nmd.2007.11.010

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


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  7 in total

1.  Muscle fat-fraction and mapping in Duchenne muscular dystrophy: evaluation of disease distribution and correlation with clinical assessments. Preliminary experience.

Authors:  Michele Gaeta; Sonia Messina; Achille Mileto; Gian Luca Vita; Giorgio Ascenti; Sergio Vinci; Antonio Bottari; Giuseppe Vita; Nicola Settineri; Daniele Bruschetta; Sergio Racchiusa; Fabio Minutoli
Journal:  Skeletal Radiol       Date:  2011-11-10       Impact factor: 2.199

2.  Therapeutic exon skipping for dysferlinopathies?

Authors:  Annemieke Aartsma-Rus; Kavita H K Singh; Ivo F A C Fokkema; Ieke B Ginjaar; Gert-Jan van Ommen; Johan T den Dunnen; Silvère M van der Maarel
Journal:  Eur J Hum Genet       Date:  2010-02-10       Impact factor: 4.246

Review 3.  Progress in therapeutic antisense applications for neuromuscular disorders.

Authors:  Annemieke Aartsma-Rus; Gert-Jan B van Ommen
Journal:  Eur J Hum Genet       Date:  2009-10-07       Impact factor: 4.246

4.  Genotype and phenotype characterization in a large dystrophinopathic cohort with extended follow-up.

Authors:  Francesca Magri; Alessandra Govoni; Maria Grazia D'Angelo; Roberto Del Bo; Serena Ghezzi; Gandossini Sandra; Anna Carla Turconi; Monica Sciacco; Patrizia Ciscato; Andreina Bordoni; Silvana Tedeschi; Francesco Fortunato; Valeria Lucchini; Sara Bonato; Costanza Lamperti; Domenico Coviello; Yvan Torrente; Stefania Corti; Maurizio Moggio; Nereo Bresolin; Giacomo Pietro Comi
Journal:  J Neurol       Date:  2011-03-12       Impact factor: 4.849

5.  Muscle histology vs MRI in Duchenne muscular dystrophy.

Authors:  M Kinali; V Arechavala-Gomeza; S Cirak; A Glover; M Guglieri; L Feng; K G Hollingsworth; D Hunt; H Jungbluth; H P Roper; R M Quinlivan; J A Gosalakkal; S Jayawant; A Nadeau; L Hughes-Carre; A Y Manzur; E Mercuri; J E Morgan; V Straub; K Bushby; C Sewry; M Rutherford; F Muntoni
Journal:  Neurology       Date:  2011-01-25       Impact factor: 9.910

6.  Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.

Authors:  Maria Kinali; Virginia Arechavala-Gomeza; Lucy Feng; Sebahattin Cirak; David Hunt; Carl Adkin; Michela Guglieri; Emma Ashton; Stephen Abbs; Petros Nihoyannopoulos; Maria Elena Garralda; Mary Rutherford; Caroline McCulley; Linda Popplewell; Ian R Graham; George Dickson; Matthew J A Wood; Dominic J Wells; Steve D Wilton; Ryszard Kole; Volker Straub; Kate Bushby; Caroline Sewry; Jennifer E Morgan; Francesco Muntoni
Journal:  Lancet Neurol       Date:  2009-08-25       Impact factor: 44.182

7.  Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.

Authors:  Jerry R Mendell; Nathalie Goemans; Linda P Lowes; Lindsay N Alfano; Katherine Berry; James Shao; Edward M Kaye; Eugenio Mercuri
Journal:  Ann Neurol       Date:  2016-01-08       Impact factor: 10.422
  7 in total

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