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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Evaluation of the Lipid‑Binding Properties of Recombinant Dystrophin Spectrin‑Like Repeat Domains R1‑3
Authors:
Cooper-Olson G, Rodino-Klapac LR, Potter RA
Journal:
Journal of Neuromuscular Diseases
Published:
23 March 2021
Comparison of Long-Term Ambulatory Function in Patients With Duchenne Muscular Dystrophy Treated With Eteplirsen and Matched Natural History Controls
Authors:
Mendell JR, Khan N, Sha N, Eliopoulos H, McDonald CM, Goemans N, Mercuri E, Lowes LP, Alfano LN, on behalf of the Eteplirsen Study Group
Journal:
Journal of Neuromuscular Diseases
Published:
19 February 2021
The Administration of Antisense Oligonucleotide Golodirsen Reduces Pathological Regeneration in Patients With Duchenne Muscular Dystrophy
Authors:
Scaglioni D, Catapano F, Ellis M, Torelli S, Chambers D, Feng L, Beck M, Sewry C, Monforte M, Harriman S, Koenig E, Malhotra J, Popplewell L, Guglieri M, Straub V, Mercuri E, Servais L, Phadke R, Morgan J, Muntoni F
Journal:
Acta Neuropathologica Communications
Published:
06 January 2021
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy
Authors:
Potter RA, Griffin DA, Heller KN, Peterson EL, Clark EK, Mendell JR, Rodino-Klapac LR
Journal:
Human Gene Therapy
Published:
04 January 2021
Assessment of Request permission From owner D6 rAAVrh74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy
Authors:
Willcocks RJ, Forbes SC, Walter GA, Sweeney L, Rodino-Klapac LR, Mendell JR, Vandenborne K
Journal:
JAMA Network Open
Published:
04 January 2021
Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy
Authors:
Griffin DA, Pozsgai ER, Heller KN, Potter RA, Peterson EL, Rodino-Klapac LR
Journal:
Human Gene Therapy
Published:
21 December 2020