Publications Library
Explore Sarepta’s library to find peer-reviewed publications by therapeutic area or program from 2018 onward.
The publications below are provided to support scientific exchange.
Publications may include information about investigational use(s) of compounds/products that
are not approved for use by the U.S. Food and Drug Administration (FDA) and/or are inconsistent
with the Prescribing Information. Sarepta does not recommend or suggest the use of any of its
products beyond the approved Prescribing Information.
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Long-Term Treatment With Eteplirsen in Nonambulatory Patients With Duchenne Muscular Dystrophy
Authors:
Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, Rodino-Klapac LR, Sahenk Z, Schnell FJ, Young GD, Mendell JR, Lowes LP
Journal:
Medicine (Baltimore)
Published:
01 June 2019
Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Authors:
Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM, Eteplirsen Investigators and the CINRG DNHS Investigators.
Journal:
Journal of Neuromuscular Diseases
Published:
07 March 2019
Validation of a Muscle-Specific Tissue Image Analysis Tool for Quantitative Assessment of Dystrophin Staining in Frozen Muscle Biopsies
Authors:
Aeffner F, Faelan C, Moore SA, Moody A, Black JC, Charleston JS, Frank DE, Dworzak J, Piper JK, Ranjitkar M, Wilson K, Kanaly S, Rudmann DG, Lange H, Young GD, Milici AJ
Journal:
Archives of Pathology & Laboratory Medicine
Published:
31 August 2018
Eteplirsen Treatment for Duchenne Muscular Dystrophy: Exon Skipping and Dystrophin Production
Authors:
Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR
Journal:
Neurology
Published:
12 June 2018
Machine Learning to Predict Cell-Penetrating Peptides for Antisense Delivery
Graphical Abstract
Authors:
Wolfe JM, Fadzen CM, Choo ZN, Holden RL, Yao M, Hanson GJ, Pentelute BL
Journal:
ACS Central Science
Published:
05 April 2018
Perfluoroaryl Bicyclic Cell-Penetrating Peptides for Delivery of Antisense Oligonucleotides
Graphical Abstract
Authors:
Wolfe JM, Fadzen CM, Holden RL, Yao M, Hanson GJ, Pentelute BL
Journal:
Angewandte Chemie International Edition
Published:
25 February 2018