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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Characterizing the Quality-of-Life Impact of Duchenne Muscular Dystrophy on Caregivers: a Case-Control Investigation
Authors:
Schwartz CE, Stark RB, Audhya IF, Gooch KL
Journal:
Journal of Patient-Reported Outcomes
Published:
20 November 2021
Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: a First-In-Human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial
Authors:
Servais L, Mercuri E, Straub V, Guglieri M, Seferian AM, Scoto M, Leone D, Koenig E, Khan N, Dugar A, Wang X, Han B, Wang D, Muntoni F, SKIP-NMD Study Group
Journal:
Nucleic Acid Therapeutics
Published:
17 November 2021
Antisense Oligonucleotides: Absorption, Distribution, Metabolism, and Excretion
Authors:
Shadid M, Badawi M, Abulrob A
Journal:
Expert Opinion on Drug Metabolism & Toxicology
Published:
22 October 2021
Pharmacokinetic/Pharmacodynamic Modeling of a Cell-Penetrating Peptide Phosphorodiamidate Morpholino Oligomer in Mdx Mice
Authors:
Mukashyaka MC, Wu CL, Ha K, Zhang J, Wood J, Foley S, Mastis B, Jungels N, Sun H, Shadid M, Harriman S, Hadcock JR
Journal:
Pharmaceutical Research
Published:
20 October 2021
Characterizing Demographics, Comorbidities and Costs of Care Among Populations With Duchenne Muscular Dystrophy With Medicaid and Commercial Coverage
Authors:
Klimchak AC, Szabo SM, Qian C, Popoff E, Iannaccone S, Gooch KL
Journal:
Journal of Managed Care & Specialty Pharmacy
Published:
01 October 2021
Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy
Authors:
Landfeldt E, Iff J, Henricson EK
Journal:
Value in Health
Published:
09 September 2021