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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Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: a Systematic Review
Authors:
Waldrop MA, Yaou RB, Lucas KK, Martin AS, O'Rourke E: FILNEMUS, Ferlini A, Muntoni F, Leturcq F, Tuffery-Giraud S, Weiss RB, Flanigan KM
Journal:
Journal of Neuromuscular Diseases
Published:
15 May 2020
Splice Modulating Antisense Oligonucleotides Restore Some Acid-Alpha-Glucosidase Activity in Cells Derived From Patients With Late-Onset Pompe Disease
Authors:
Aung-Htut MT, Ham KA, Tchan M, Johnsen R, Schnell FJ, Fletcher S, Wilton SD
Journal:
Scientific Reports
Published:
21 April 2020
Increased Dystrophin Production With Golodirsen in Patients With Duchenne Muscular Dystrophy
Authors:
Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F, SKIP-NMD Study Group
Journal:
Neurology
Published:
05 March 2020
Clinical Development on the Frontier: Gene Therapy for Duchenne Muscular Dystrophy
Authors:
Asher DR, Thapa K, Dharia SD, Khan N, Potter RA, Rodino-Klapac LR, Mendell JR
Journal:
Expert Opinion on Biological Therapy
Published:
12 February 2020
Chimeras of Cell-Penetrating Peptides Demonstrate Synergistic Improvement in Antisense Efficacy
Graphical Abstract
Authors:
Fadzen CM, Holden RL, Wolfe JM, Choo ZN, Schissel CK, Yao M, Hanson GJ, Pentelute BL
Journal:
Biochemistry
Published:
26 August 2019
Adapting Traditional Content Validation Methods to Fit Purpose: an Example With a Novel Video Assessment and Training Materials in Duchenne Muscular Dystrophy (DMD)
Authors:
White MK, Leffler M, Rychlec K, Jones C, McSherry C, Walker L, Kosinski M
Journal:
Quality of Life Research
Published:
13 July 2019