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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Survival Among Patients Receiving Eteplirsen for up to 8 Years for the Treatment of Duchenne Muscular Dystrophy and Contextualization With Natural History Controls
Authors:
Iff J, Done N, Tuttle E, Zhong Y, Wei F, Darras BT, McDonald CM, Mercuri E, Muntoni F
Journal:
Muscle & Nerve
Published:
14 March 2024
Use of Plasmapheresis to Lower Anti-Aav Antibodies in Nonhuman Primates With Pre-Existing Immunity to Aavrh74
Graphical Abstract
Authors:
Potter RA, Peterson EL, Griffin D, Cooper Olson G, Lewis S, Cochran K, Mendell JR, Rodino-Klapac LR
Journal:
Molecular Therapy Methods & Clinical Development
Published:
14 March 2024
Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients With Single Exon Duplications: an Open-Label Study
Authors:
Nicolau S, Malhotra J, Kaler M, Magistrado-Coxen P, Iammarino MA, Reash NF, Frair EC, Wijeratne S, Kelly BJ, White P, Lowes LP, Waldrop MA, Flanigan KM
Journal:
Journal of Neuromuscular Diseases
Published:
08 March 2024
Comparing Preferences for Disease Profiles: a Discrete Choice Experiment With a US Societal Perspective
Authors:
Johnston KM, Audhya IF, Dunne J, Feeny D, Neumann P, Malone DC, Szabo SM, Gooch KL
Journal:
Applied Health Economics and Health Policy
Published:
23 January 2024
Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy
Authors:
Mendell JR, Proud C, Zaidman CM, Mason S, Darton E, Wang S, Wandel C, Murphy AP, Mercuri E, Muntoni F, McDonald CM
Journal:
Pediatric Neurology
Published:
05 January 2024
Gene Therapy With Bidridistrogene Xeboparvovec for Limb-Girdle Muscular Dystrophy Type 2E/R4: Phase 1/2 Trial Results
Authors:
Mendell JR, Pozsgai ER, Lewis S, Griffin DA, Lowes LP, Alfano LN, Lehman KJ, Church K, Reash NF, Iammarino MA, Sabo B, Potter R, Neuhaus S, Li X, Stevenson H, Rodino-Klapac LR
Journal:
Nature Medicine
Published:
04 January 2024