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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In-Cell Penetration Selection-Mass Spectrometry Produces Noncanonical Peptides for Antisense Delivery
Graphical Abstract
Authors:
Schissel CK, Farquhar CE, Loas A, Malmberg AB, Pentelute BL
Journal:
American Chemical Society (ACS) Chemical Biology
Published:
17 March 2023
Concept Elicitation Interviews and Conceptual Model to Understand the Patient Experience of Limb Girdle Muscular Dystrophy
Authors:
Johnston K, Casstevens C, Patel VP, Merikle E, Presnall C, Audhya I
Journal:
Advances in Therapy
Published:
14 March 2023
Therapeutic Phosphorodiamidate Morpholino Oligonucleotides: Physical Properties, Solution Structures, and Folding Thermodynamics
Graphical Abstract
Authors:
Maksudov F, Kliuchnikov E, Pierson D, Ujwal ML, Marx KA, Chanda A, Barsegov V
Journal:
Molecular Therapy Nucleic Acids
Published:
14 March 2023
Commercial Health Plans Use of Patient Subgroup Restrictions: an Analysis of Orphan and Fda-Expedited Programs
Plain Language Summary
Authors:
Jenkins NB, Rucker JA, Klimchak AC, Sedita LE, Chambers JD
Journal:
Journal of Managed Care & Specialty Pharmacy
Published:
02 March 2023
Evaluation of Raavrh74 Gene Therapy Vector Seroprevalence by Measurement of Total Binding Antibodies in Patients With Duchenne Muscular Dystrophy
Authors:
Goedeker NL, Dharia SD, Griffin DA, Coy J, Truesdale T, Parikh R, Whitehouse K, Santra S, Asher DR, Zaidman CM
Journal:
Therapeutic Advances in Neurological Disorders
Published:
24 January 2023
Systemic Îł-Sarcoglycan AAV Gene Transfer Results in Dose-Dependent Correction of Muscle Deficits in the LGMD 2C/R5 Mouse Model
Graphical Abstract
Authors:
Seo YE, Baine SH, Kempton AN, Rogers OC, Lewis S, Adegboye K, Haile A, Griffin DA, Peterson EL, Pozsgai ER, Potter RA, Rodino-Klapac LR
Journal:
Molecular Therapy Methods & Clinical Development
Published:
16 January 2023