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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A Cell-Penetrating Peptide Enhances Delivery and Efficacy of Phosphorodiamidate Morpholino Oligomers in Animals (RC-1001)
Graphical Abstract
Authors:
Gan L, Wood JA, Yao M, Treleaven CM, Estrella NL, Passini MA, Wentworth BM, Hanson GJ, Wu CL
Journal:
Molecular Therapy – Nucleic Acids
Published:
13 December 2022
Characterizing the Occurrence of Key Clinical Milestones in Duchenne Muscular Dystrophy in the United States Using Real-World Data
Authors:
Szabo SM, Klimchak AC, Qian C, Iannaccone S, Popoff E, Gooch KL
Journal:
Journal of Neuromuscular Diseases
Published:
08 October 2022
Patient Life Aspirations in the Context of Duchenne Muscular Dystrophy: a Mixed-Methods Case-Control Study
Authors:
Schwartz CE, Biletch E, Stuart RBB, Rapkin BD
Journal:
Journal of Patient-Reported Outcomes
Published:
14 September 2022
Qalys and Ambulatory Status: Societal Preferences for Healthcare Decision Making
Authors:
Freath LL, Curry AS, Cork DMW, Audhya IF, Gooch KL
Journal:
Journal of Medical Economics
Published:
28 June 2022
Delays in Pulmonary Decline in Eteplirsen-Treated Patients With Duchenne Muscular Dystrophy
Authors:
Iff J, Gerrits C, Zhong Y, Tuttle E, Brik E, Zheng Y, Paul X, Henricson EK, McDonald CM, CINRG-DNHS Investigators
Journal:
Muscle & Nerve
Published:
18 June 2022
Factors Associated With the Health-Related Quality of Life Among People With Duchenne Muscular Dystrophy: a Study Using the Health Utilities Index (HUI)
Authors:
Szabo SM, Audhya IF, Rogula B, Feeny D, Gooch KL
Journal:
Health and Quality of Life Outcomes
Published:
11 June 2022