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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Safety, Tolerability and Pharmacokinetics of Eteplirsen in Young Boys Aged 6 to 48 Months With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Authors:
Mercuri E, Seferian AM, Servais L, Deconinck N, Stevenson H, Ni X, Zhang W, East L, Yonren S, Muntoni F, 4658-102 Study Group
Journal:
Neuromuscular Disorders
Published:
01 June 2023
Assessing the Value of Delandistrogene Moxeparvovec (SRP-9001) Gene Transfer Therapy in Patients With Duchenne Muscular Dystrophy
Authors:
Klimchak AC, Sedita LE, Rodino-Klapac LR, Mendell JR, McDonald CM, Gooch KL, Malone DC
Journal:
Journal of Market Access & Health Policy
Published:
26 May 2023
Medicaid Coverage Practices for Approved Gene and Cell Therapies: Existing Barriers and Proposed Policy Solutions
Graphical Abstract
Authors:
Allen J, Berry D, Cook F, Hume A, Rouce R, Srirangam A, Wellman J, McCombs C
Journal:
Molecular Therapy Methods & Clinical Development
Published:
16 May 2023
Assessing the Impact of Single or Short-Term Administration on a Therapy’s Cost-Effectiveness: a Hypothetical Disease-Agnostic Model
Authors:
Klimchak AC, Sedita LE, Gooch KL, Malone DC
Journal:
Journal of Medical Economics
Published:
23 April 2023
Assessing the Impact of Single or Short-Term Administration on a Therapy's Cost-Effectiveness: a Hypothetical Disease-Agnostic Model
Authors:
Klimchak AC, Sedita LE, Gooch KL, Malone DC
Journal:
Journal of Medical Economics
Published:
23 April 2023
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