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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DMD Antisense Oligonucleotide Mediated Exon Skipping Efficiency Correlates With Flanking Intron Retention Time and Target Position Within the Exon
Authors:
Goossens R, Verwey N, Ariyurek Y, Schnell F, Aartsma-Rus A
Journal:
RNA Biology
Published:
04 September 2023
Real-World Evidence of Eteplirsen Treatment Effects in Patients With Duchenne Muscular Dystrophy in the USA
Plain Language Summary
Authors:
Iff J, Zhong Y, Tuttle E, Gupta D, Paul X, Henricson E
Journal:
Journal of Comparative Effectiveness Research
Published:
23 August 2023
Long-Term Safety and Functional Outcomes of Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy: a Phase 1/2A Nonrandomized Trial
Authors:
Mendell JR, Sahenk Z, Lehman KJ, Lowes LP, Reash NF, Iammarino MA, Alfano LN, Lewis S, Church K, Shell R, Potter RA, Griffin DA, Hogan M, Wang S, Mason S, Darton E, Rodino-Klapac LR
Journal:
Muscle & Nerve
Published:
14 August 2023
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) With Duchenne Muscular Dystrophy: 1-Year Interim Results From Study SRP-9001-103 (ENDEAVOR)
Authors:
Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR
Journal:
Annals of Neurology
Published:
04 August 2023
Exploring the Relationship Between North Star Ambulatory Assessment and Health Utilities Index Scores in Duchenne Muscular Dystrophy
Authors:
Audhya I, Rogula B, Szabo SM, Feeny D, Bolatova T, Gooch K
Journal:
Health and Quality of Life Outcomes
Published:
19 July 2023
Expression of SRP-9001 Dystrophin and Stabilization of Motor Function up to 2 Years Post-Treatment With Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy
Authors:
Mendell JR, Shieh PB, McDonald CM, Sahenk Z, Lehman KJ, Lowes LP, Reash NF, Iammarino MA, Alfano LN, Sabo B, Woods JD, Skura CL, Mao HC, Staudt LA, Griffin DA, Lewis S, Wang S, Potter RA, Singh T, Rodino-Klapac LR
Journal:
Frontiers in Cell and Developmental Biology
Published:
10 July 2023