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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Unmet Needs and Evolving Treatment for Limb Girdle Muscular Dystrophies
Plain Language Summary
Authors:
Pozsgai E, Griffin D, Potter R, Sahenk Z, Lehman K, Rodino-Klapac LR, Mendell JR
Journal:
Neurodegenerative Disease Management
Published:
02 September 2021
A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline With Long-Term Eteplirsen Treatment
Authors:
Mitelman O, Abdel-Hamid HZ, Byrne BJ, Connolly AM, Heydemann P, Proud C, Shieh PB, Wagner KR, Dugar A, Santra S, Signorovitch J, Goemans N for the investigators from the LNMRC Natural History study, McDonald CM for the investigators from the CINRG Duchenne National History Study, Mercuri E for the investigators from The DMD Italian Group, Mendell JR
Journal:
Journal of Neuromuscular Diseases
Published:
19 August 2021
Safety, Tolerability, and Pharmacokinetics of Casimersen in Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping: a Phase 1/2 Randomized, Double-Blind, Placebo-Controlled, Dose-Titration Trial
Authors:
Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB
Journal:
Muscle & Nerve
Published:
08 June 2021
Open-Label Evaluation of Eteplirsen in Male Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Vs Untreated Control: PROMOVI Trial
Authors:
McDonald CM, Shieh PB, Abdel-Hamid HZ, Connolly AM, Ciafaloni E, Wagner KR, Goemans N, Mercuri E, Khan N, Koenig E, Malhotra J, Zhang W, Han B, Mendell JR, the Italian DMD Telethon Registry Study Group, Leuven NMRC Registry Investigators, CINRG Duchenne Natural History Investigators, and PROMOVI Trial Clinical Investigators
Journal:
Journal of Neuromuscular Diseases
Published:
08 June 2021
The Clinical Course of Duchenne Muscular Dystrophy in the Corticosteroid Treatment Era: a Systematic Literature Review
Authors:
Szabo SM, Salhany RM, Deighton A, Harwood M, Mah J, Gooch KL
Journal:
Orphanet Journal of Rare Diseases
Published:
22 May 2021
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