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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Estimating Patient-Reported Health State Utilities in Duchenne Muscular Dystrophy Using the Health Utilities Index and EQ-5D
Authors:
Audhya IF, Szabo SM, Bever A, O'Sullivan F, Malone DC, Feeny D, Neumann P, Iannaccone ST, Jayasinghe P, Gooch KL
Journal:
Journal of Patient‑Reported Outcomes
Published:
15 December 2023
A Qualitative Study to Understand the Duchenne Muscular Dystrophy Experience From the Parent/Patient Perspective
Authors:
Brown V, Merikle E, Johnston K, Gooch K, Audhya I, Lowes L
Journal:
Journal of Patient‑Reported Outcomes
Published:
12 December 2023
Aav-Based Crispr-Cas9 Genome Editing: Challenges and Engineering Opportunities
Authors:
Kabadi AM, Mejia-Guerra MK, Graef JD, Khan SZ, Walton EM, Wang X, Gersbach CA, Potter R
Journal:
Current Opinion in Biomedical Engineering
Published:
07 December 2023
Patterns of Clinical Progression Among Patients With Autosomal Recessive Limb-Girdle Muscular Dystrophy: a Systematic Review
Authors:
Cheung A, Audhya IF, Szabo SM, Friesen M, Weihl CC, Gooch KL
Journal:
Journal of Clinical Neuromuscular Disease
Published:
01 December 2023
Expression and Function of Four Aav-Based Constructs for Dystrophin Restoration in the Mdx Mouse Model of Duchenne Muscular Dystrophy
Authors:
Potter RA, Griffin DA, Heller KN, Mendell JR, Rodino-Klapac LR
Journal:
Biology Open
Published:
18 September 2023
Paving the Way for Future Gene Therapies: a Case Study of Scientific Spillover From Delandistrogene Moxeparvovec
Graphical Abstract
Authors:
Asher D, Dai D, Klimchak AC, Sedita LE, Gooch KL, Rodino-Klapac L
Journal:
Molecular Therapy – Methods & Clinical Development
Published:
14 September 2023