Literature DB >> 27916994

Eteplirsen Approved for Duchenne Muscular Dystrophy: The FDA Faces a Difficult Choice.

Cy A Stein1.   

Abstract

Entities:  

Mesh：

Substances：

Year:  2016        PMID： 27916994      PMCID： PMC5154484          DOI： 10.1038/mt.2016.188

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


× No keyword cloud information.
  6 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.  Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.

Authors:  Sebahattin Cirak; Virginia Arechavala-Gomeza; Michela Guglieri; Lucy Feng; Silvia Torelli; Karen Anthony; Stephen Abbs; Maria Elena Garralda; John Bourke; Dominic J Wells; George Dickson; Matthew J A Wood; Steve D Wilton; Volker Straub; Ryszard Kole; Stephen B Shrewsbury; Caroline Sewry; Jennifer E Morgan; Kate Bushby; Francesco Muntoni
Journal:  Lancet       Date:  2011-07-23       Impact factor: 79.321

Review 3.  Translational and regulatory challenges for exon skipping therapies.

Authors:  Annemieke Aartsma-Rus; Alessandra Ferlini; Nathalie Goemans; Anna M G Pasmooij; Dominic J Wells; Katerine Bushby; Elizabeth Vroom; Pavel Balabanov
Journal:  Hum Gene Ther       Date:  2014-10       Impact factor: 5.695

4.  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

Review 5.  Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.

Authors:  Jacqueline N Robinson-Hamm; Charles A Gersbach
Journal:  Hum Genet       Date:  2016-08-20       Impact factor: 4.132

6.  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
  6 in total
  31 in total

Review 1.  Modulation of aberrant splicing in human RNA diseases by chemical compounds.

Authors:  Naoyuki Kataoka
Journal:  Hum Genet       Date:  2017-03-31       Impact factor: 4.132

2.  Nanoparticle Technology: Having Impact, but Needing Further Optimization.

Authors:  S Moein Moghimi; Ernst Wagner
Journal:  Mol Ther       Date:  2017-06-16       Impact factor: 11.454

3.  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 4.  FDA-Approved Oligonucleotide Therapies in 2017.

Authors:  Cy A Stein; Daniela Castanotto
Journal:  Mol Ther       Date:  2017-03-31       Impact factor: 11.454

Review 5.  Molecular Therapies for Muscular Dystrophies.

Authors:  Ava Y Lin; Leo H Wang
Journal:  Curr Treat Options Neurol       Date:  2018-06-21       Impact factor: 3.598

Review 6.  RNA therapy: Are we using the right molecules?

Authors:  Ai-Ming Yu; Chao Jian; Allan H Yu; Mei-Juan Tu
Journal:  Pharmacol Ther       Date:  2018-12-04       Impact factor: 12.310

7.  Antisense Oligonucleotide Treatment in a Humanized Mouse Model of Duchenne Muscular Dystrophy and Highly Sensitive Detection of Dystrophin Using Western Blotting.

Authors:  Rika Maruyama; Toshifumi Yokota
Journal:  Methods Mol Biol       Date:  2021

Review 8.  More than a messenger: Alternative splicing as a therapeutic target.

Authors:  A J Black; J R Gamarra; J Giudice
Journal:  Biochim Biophys Acta Gene Regul Mech       Date:  2019-07-02       Impact factor: 4.490

9.  Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.

Authors:  Yusuke Echigoya; Kenji Rowel Q Lim; Nhu Trieu; Bo Bao; Bailey Miskew Nichols; Maria Candida Vila; James S Novak; Yuko Hara; Joshua Lee; Aleksander Touznik; Kamel Mamchaoui; Yoshitsugu Aoki; Shin'ichi Takeda; Kanneboyina Nagaraju; Vincent Mouly; Rika Maruyama; William Duddy; Toshifumi Yokota
Journal:  Mol Ther       Date:  2017-07-28       Impact factor: 11.454

10.  RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges.

Authors:  Ai-Ming Yu; Young Hee Choi; Mei-Juan Tu
Journal:  Pharmacol Rev       Date:  2020-10       Impact factor: 25.468
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.