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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Disease Progression Stages and Burden in Patients With Duchenne Muscular Dystrophy Using Administrative Claims Supplemented by Electronic Medical Records
Authors:
Iff J, Zhong Y, Gupta D, Paul X, Tuttle E, Henricson E, Schrader R, CINRG DNHS Investigators
Journal:
Advances in Therapy
Published:
23 April 2022
Interplay of Disability, Caregiver Impact, and Out-Of-Pocket Expenditures in Duchenne Muscular Dystrophy: a Cohort Study
Authors:
Schwartz CE, Stark RB, Borowiec K, Audhya IF, Gooch KL
Journal:
Journal of Patient-Reported Outcomes
Published:
10 March 2022
Testing Preexisting Antibodies Prior to AAV Gene Transfer Therapy: Rationale, Lessons and Future Considerations
Graphical Abstract
Authors:
Mendell JR, Connolly AM, Lehman KJ, Griffin DA, Khan SZ, Dharia SD, Quintana-Gallardo L, Rodino-Klapac LR
Journal:
Molecular Therapy Methods & Clinical Development
Published:
26 February 2022
Societal Perspectives on Disease and Treatment Attributes Characterizing Rare Diseases: a Qualitative Study From the United States
Authors:
Szabo SM, Audhya IF, Feeny D, Neumann P, Malone DC, Gooch KL
Journal:
Journal of Patient-Reported Outcomes
Published:
24 January 2022
The Impact of Genotype on Outcomes in Individuals With Duchenne Muscular Dystrophy: a Systematic Review
Authors:
Szabo SM, Gooch KL, Mickle AT, Salhany RM, Connolly AM
Journal:
Muscle & Nerve. 022 Mar;65(3):266-277
Published:
08 December 2021
Measuring Duchenne Muscular Dystrophy Impact: Development of a Proxy-Reported Measure Derived From PROMIS Item Banks
Authors:
Schwartz CE, Stark RB, Cella D, Borowiec K, Gooch KL, Audhya IF
Journal:
Orphanet Journal of Rare Diseases
Published:
22 November 2021