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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Psychometric Evaluation of the PROMIS Parent Proxy Mobility Item Bank for Use in Duchenne Muscular Dystrophy
Authors:
Lowes LP, Le Reun CM, Alfano LN, Reash NF, Iammarino MA, Patel S, Audhya IF
Journal:
Developmental Medicine and Child Neurology
Published:
19 December 2024
Long-Term Survival and Myocardial Function Following Systemic Delivery of Delandistrogene Moxeparvovec in DMD Mdx Rats
Authors:
Baine S, Wier C, Lemmerman L, Cooper-Olson G, Kempton A, Haile A, Endres J, Fedoce A, Nesbit E, Rodino-Klapac LR, Potter RA
Journal:
Human Gene Therapy
Published:
28 November 2024
Caregiver Global Impression Observations From EMBARK: a Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients With Duchenne Muscular Dystrophy
Authors:
McDonald CM, Elkins JS, Dharmarajan S, Gooch K, Ciobanu T, Lansdall CJ, Murphy AP, McDougall F, Mercuri EM, Audhya I, EMBARK Study Group
Journal:
Neurology and Therapy
Published:
26 November 2024
Characterization of Nonclinical Drug Metabolism and Pharmacokinetic Properties of Phosphorodiamidate Morpholino Oligonucleotides, a Novel Drug Class for Duchenne Muscular Dystrophy
Graphical Abstract
Authors:
Goey AKL, Mukashyaka MC, Patel Y, Rodino-Klapac LR, East L
Journal:
Drug Metabolism and Disposition
Published:
15 November 2024
Assessment of Phosphorodiamidate Morpholino Oligomer Treatment Patterns for Patients With Duchenne Muscular Dystrophy: a Marketscan Claims Analysis
Authors:
Klimchak AC, Signorovitch J, Innis B, Laverty CG, Gooch K
Journal:
Advances in Therapy
Published:
11 November 2024
AAV Gene Therapy for Duchenne Muscular Dystrophy: the EMBARK Phase 3 Randomized Trial
Authors:
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR
Journal:
Nature Medicine
Published:
09 October 2024