Literature DB >> 27857122

Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line.

James J Dowling1.   

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Year:  2016        PMID： 27857122     DOI： 10.1038/nrneurol.2016.180

Source DB:  PubMed          Journal:  Nat Rev Neurol        ISSN： 1759-4758            Impact factor:   42.937


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  5 in total

1.  Eteplirsen for the treatment of Duchenne muscular dystrophy.

Authors:  Jerry R Mendell; Louise R Rodino-Klapac; Zarife Sahenk; Kandice Roush; Loren Bird; Linda P Lowes; Lindsay Alfano; Ann Maria Gomez; Sarah Lewis; Janaiah Kota; Vinod Malik; Kim Shontz; Christopher M Walker; Kevin M Flanigan; Marco Corridore; John R Kean; Hugh D Allen; Chris Shilling; Kathleen R Melia; Peter Sazani; Jay B Saoud; Edward M Kaye
Journal:  Ann Neurol       Date:  2013-09-10       Impact factor: 10.422

2.  Revertant fibres: a possible genetic therapy for Duchenne muscular dystrophy?

Authors:  S D Wilton; D E Dye; L M Blechynden; N G Laing
Journal:  Neuromuscul Disord       Date:  1997-07       Impact factor: 4.296

Review 3.  Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management.

Authors:  Katharine Bushby; Richard Finkel; David J Birnkrant; Laura E Case; Paula R Clemens; Linda Cripe; Ajay Kaul; Kathi Kinnett; Craig McDonald; Shree Pandya; James Poysky; Frederic Shapiro; Jean Tomezsko; Carolyn Constantin
Journal:  Lancet Neurol       Date:  2009-11-27       Impact factor: 44.182

4.  Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study.

Authors:  Thomas Voit; Haluk Topaloglu; Volker Straub; Francesco Muntoni; Nicolas Deconinck; Giles Campion; Sjef J De Kimpe; Michelle Eagle; Michela Guglieri; Steve Hood; Lia Liefaard; Afrodite Lourbakos; Allison Morgan; Joanna Nakielny; Naashika Quarcoo; Valeria Ricotti; Katie Rolfe; Laurent Servais; Claire Wardell; Rosamund Wilson; Padraig Wright; John E Kraus
Journal:  Lancet Neurol       Date:  2014-09-07       Impact factor: 44.182

5.  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
  5 in total
  17 in total

Review 1.  The role of RNA alternative splicing in regulating cancer metabolism.

Authors:  Itamar Kozlovski; Zahava Siegfried; Adi Amar-Schwartz; Rotem Karni
Journal:  Hum Genet       Date:  2017-04-20       Impact factor: 4.132

2.  Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy.

Authors:  Leonela Amoasii; Chengzu Long; Hui Li; Alex A Mireault; John M Shelton; Efrain Sanchez-Ortiz; John R McAnally; Samadrita Bhattacharyya; Florian Schmidt; Dirk Grimm; Stephen D Hauschka; Rhonda Bassel-Duby; Eric N Olson
Journal:  Sci Transl Med       Date:  2017-11-29       Impact factor: 17.956

Review 3.  Using Genome Sequence to Enable the Design of Medicines and Chemical Probes.

Authors:  Alicia J Angelbello; Jonathan L Chen; Jessica L Childs-Disney; Peiyuan Zhang; Zi-Fu Wang; Matthew D Disney
Journal:  Chem Rev       Date:  2018-01-11       Impact factor: 60.622

4.  Androgen receptor agonists increase lean mass, improve cardiopulmonary functions and extend survival in preclinical models of Duchenne muscular dystrophy.

Authors:  Suriyan Ponnusamy; Ryan D Sullivan; Dahui You; Nadeem Zafar; Chuan He Yang; Thirumagal Thiyagarajan; Daniel L Johnson; Maron L Barrett; Nikki J Koehler; Mayra Star; Erin J Stephenson; Dave Bridges; Stephania A Cormier; Lawrence M Pfeffer; Ramesh Narayanan
Journal:  Hum Mol Genet       Date:  2017-07-01       Impact factor: 6.150

5.  Mitochondrial Targeting Probes, Drug Conjugates, and Gene Therapeutics.

Authors:  Carmine Pasquale Cerrato; Tove Kivijärvi; Ülo Langel
Journal:  Methods Mol Biol       Date:  2022

Review 6.  Animal models for researching approaches to therapy of Duchenne muscular dystrophy.

Authors:  M I Zaynitdinova; A V Lavrov; S A Smirnikhina
Journal:  Transgenic Res       Date:  2021-08-18       Impact factor: 2.788

7.  Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy.

Authors:  Leonela Amoasii; John C W Hildyard; Hui Li; Efrain Sanchez-Ortiz; Alex Mireault; Daniel Caballero; Rachel Harron; Thaleia-Rengina Stathopoulou; Claire Massey; John M Shelton; Rhonda Bassel-Duby; Richard J Piercy; Eric N Olson
Journal:  Science       Date:  2018-08-30       Impact factor: 47.728

Review 8.  Progress toward Gene Therapy for Duchenne Muscular Dystrophy.

Authors:  Joel R Chamberlain; Jeffrey S Chamberlain
Journal:  Mol Ther       Date:  2017-04-15       Impact factor: 12.910

9.  Targeting KIT by frameshifting mRNA transcripts as a therapeutic strategy for aggressive mast cell neoplasms.

Authors:  Douglas B Snider; Greer K Arthur; Guido H Falduto; Ana Olivera; Lauren C Ehrhardt-Humbert; Emmaline Smith; Cierra Smith; Dean D Metcalfe; Glenn Cruse
Journal:  Mol Ther       Date:  2021-08-08       Impact factor: 11.454

Review 10.  Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing.

Authors:  Britt Hanson; Matthew J A Wood; Thomas C Roberts
Journal:  RNA Biol       Date:  2021-01-20       Impact factor: 4.652
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