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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Household Costs in the United States for Accommodating Functional Impairments Associated With Duchenne Muscular Dystrophy: Results From a Caregiver Survey
Authors:
Innis B, Jarvis J, Renteria T, Audhya I
Journal:
Orphanet Journal of Rare Diseases
Published:
12 June 2025
Splicing Correction by Peptide-Conjugated Morpholinos as a Novel Treatment for Late-Onset Pompe Disease
Graphical Abstract
Authors:
Oliver RA, Ahern ME, Castaneda PG, Jinadasa T, Bardhan A, Morgan KY, Ha K, Adhikari K, Jungels N, Liberman N, Mitra A, Greer CD, Wright AM, Thompson EG, Garcia S, Copson E, Allu S, Tan X, Callahan AJ, Cai BZ, Guerlavais V, Kim KJ, Malmberg AB
Journal:
Molecular Therapy Nucleic Acids
Published:
27 March 2025
A Population Pharmacokinetic Model to Inform Extension of the Eteplirsen Dosing Regimen Across the Broad DMD Population
Authors:
Patel Y, Orogun L, Yocum N, Rodino-Klapac LR, East L
Journal:
CPT: Pharmacometrics & Systems Pharmacology
Published:
26 February 2025
Longitudinal Outcomes Among Patients With Duchenne Muscular Dystrophy: a Canadian Retrospective Population-Based Study
Authors:
Qian C, Klimchak AC, Szabo SM, Gooch KL, Dragan R, Prior HJ, Mah JK
Journal:
Muscle & Nerve
Published:
13 February 2025
Quantifying the Altruism Value for a Rare Pediatric Disease: Duchenne Muscular Dystrophy
Authors:
Shafrin J, Thahir S, Klimchak AC, Audhya I, Sedita LE, Romley JA
Journal:
The American Journal of Managed Care
Published:
14 January 2025
Immunologic Investigations Into Transgene Directed Immune-Mediated Myositis Following Delandistrogene Moxeparvovec Gene Therapy
Authors:
Potter RA, Moeller IH, Khan S, Haegel H, Hollenstein A, Steiner G, Wandel C, Murphy AP, Asher DR, Palatinsky E, Griffin DA, Mason S, Iannaccone ST, Zaidman CM, Rodino-Klapac LR
Journal:
Scientific Reports
Published:
02 January 2025