Literature DB >> 28515510

Eteplirsen.

Danial E Baker.   

Abstract

Each month, subscribers to The Formulary Monograph Service receive 5 to 6 well-documented monographs on drugs that are newly released or are in late phase 3 trials. The monographs are targeted to Pharmacy & Therapeutics Committees. Subscribers also receive monthly 1-page summary monographs on agents that are useful for agendas and pharmacy/nursing in-services. A comprehensive target drug utilization evaluation/medication use evaluation (DUE/MUE) is also provided each month. With a subscription, the monographs are available online to subscribers. Monographs can be customized to meet the needs of a facility. Through the cooperation of The Formulary, Hospital Pharmacy publishes selected reviews in this column. For more information about The Formulary Monograph Service, contact Wolters Kluwer customer service at 866-397-3433. The April 2017 monograph topics are deflazacort, plecanatide, delafloxacin, oxymetazoline hydrochloride 1% cream, and betrixaban. The DUE is on plecanatide.

Entities:  

Year:  2017        PMID： 28515510      PMCID： PMC5424835          DOI： 10.1310/hpj5204-302

Source DB:  PubMed          Journal:  Hosp Pharm        ISSN： 0018-5787


  11 in total

Review 1.  Pharmacological prospects in the treatment of Duchenne muscular dystrophy.

Authors:  Urs T Ruegg
Journal:  Curr Opin Neurol       Date:  2013-10       Impact factor: 5.710

2.  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

3.  Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy.

Authors:  Sebahattin Cirak; Lucy Feng; Karen Anthony; Virginia Arechavala-Gomeza; Silvia Torelli; Caroline Sewry; Jennifer E Morgan; Francesco Muntoni
Journal:  Mol Ther       Date:  2011-11-15       Impact factor: 11.454

Review 4.  Exon skipping therapy for Duchenne muscular dystrophy.

Authors:  Ryszard Kole; Arthur M Krieg
Journal:  Adv Drug Deliv Rev       Date:  2015-05-14       Impact factor: 15.470

5.  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

6.  Exon skipping quantification by quantitative reverse-transcription polymerase chain reaction in Duchenne muscular dystrophy patients treated with the antisense oligomer eteplirsen.

Authors:  Karen Anthony; Lucy Feng; Virginia Arechavala-Gomeza; Michela Guglieri; Volker Straub; Katherine Bushby; Sebahattin Cirak; Jennifer Morgan; Francesco Muntoni
Journal:  Hum Gene Ther Methods       Date:  2012-10       Impact factor: 2.396

7.  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

8.  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

Review 9.  Current and emerging treatment strategies for Duchenne muscular dystrophy.

Authors:  Jean K Mah
Journal:  Neuropsychiatr Dis Treat       Date:  2016-07-22       Impact factor: 2.570

10.  Dystromirs as serum biomarkers for monitoring the disease severity in Duchenne muscular Dystrophy.

Authors:  Irina T Zaharieva; Mattia Calissano; Mariacristina Scoto; Mark Preston; Sebahattin Cirak; Lucy Feng; James Collins; Ryszard Kole; Michela Guglieri; Volker Straub; Kate Bushby; Alessandra Ferlini; Jennifer E Morgan; Francesco Muntoni
Journal:  PLoS One       Date:  2013-11-25       Impact factor: 3.240
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  2 in total

1.  Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice.

Authors:  Li Xu; Yandi Gao; Yeh Siang Lau; Renzhi Han
Journal:  J Vis Exp       Date:  2018-08-02       Impact factor: 1.355

Review 2.  Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot.

Authors:  Harry Wilton-Clark; Toshifumi Yokota
Journal:  Genes (Basel)       Date:  2022-01-28       Impact factor: 4.096
  2 in total

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