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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The EQ-5D and Health Utilities Index for Assessing Health-Related Quality-of-Life Impact in Duchenne Muscular Dystrophy: Evaluating the Relevance and Interpretation of Descriptive Systems From Patient and Caregiver Perspectives
Authors:
Szabo SM, Griffin E, Iannaccone ST, Gooch KL, Audhya IF
Journal:
Advances in Patient-Reported Outcomes
Published:
09 November 2025
Caregiver-Reported Patient Experiences With Duchenne Muscular Dystrophy: Qualitative In-Trial Interviews 1 Year After Delandistrogene Moxeparvovec in the Pivotal EMBARK Trial
Authors:
Audhya I, Nacson AB, Gooch K, Basnyat B, Slota C, Martin S, Murphy A, Lansdall CJ, Ciobanu T, Nascimento A, Veerapandiyan A
Journal:
Neurology and Therapy
Published:
03 November 2025
Pharmacokinetic and Pharmacodynamic Evaluation of Bidridistrogene Xeboparvovec in an Aged Murine Model of Limb-Girdle Muscular Dystrophy Type 2E/R4
Authors:
Baine S, Cui J, Seo YE, Kempton A, Rogers O, Haile A, Adegboye K, Wu J, Lemmerman L, Guerrero M, Jones C, Mayes H, Freehafer T, Lewis S, Pozsgai E, East L, Rodino-Klapac LR, Potter R
Journal:
Human Gene Therapy
Published:
30 October 2025
Characterization of a Humanized Mouse Model of Duchenne Muscular Dystrophy to Support the Development of Genetic Medicines
Authors:
Braunreiter K, Kempton A, Mejia-Guerra MK, Murray A, Baine S, Adegboye K, Haile A, Ahuja SJK, Fedoce A, Liu C, Burch P, Kabadi AM
Journal:
Disease Models & Mechanisms
Published:
17 October 2025
Association Between Exon-Skipping Therapy With Eteplirsen and Cardiac Outcomes in Duchenne Muscular Dystrophy
Authors:
Iff J, Desguerre I, Liu Y, Sarkozy F, Tuttle E, Muntoni F, McDonald CM, Nougues MC, Amthor H, Zhong Y, Wahbi K, Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study Investigators
Journal:
Journal of Neuromuscular Diseases
Published:
19 August 2025
Quantitative Muscle Magnetic Resonance Outcomes in Patients With Duchenne Muscular Dystrophy: an Exploratory Analysis From the EMBARK Randomized Clinical Trial
Authors:
Vandenborne K, Walter GA, Straub V, Willcocks RJ, Forbes SC, Mercuri EM, Muntoni F, Ding K, Ennamuri S, Reid C, Murphy AP, Manfrini M, Mendell JR, Elkins JS, Rodino-Klapac LR
Journal:
JAMA Neurology
Published:
01 July 2025