Safety, tolerability, and pharmacokinetics of casimersen in patients with D uchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial-Reference-Cited by-同舟云学术

Safety, tolerability, and pharmacokinetics of casimersen in patients with D uchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial

Published:2021-06-29 Issue:3 Volume:64 Page:285-292
ISSN:0148-639X
Container-title:Muscle & Nerve
language:en
Short-container-title:Muscle & Nerve

Author:

Wagner Kathryn R.¹²,Kuntz Nancy L.³,Koenig Erica⁴,East Lilly⁵,Upadhyay Sameer⁶,Han Baoguang⁷,Shieh Perry B.⁸^ORCID

Affiliation:

1. Center for Genetic Muscle Disorders Kennedy Krieger Institute Baltimore Maryland USA

2. Departments of Neurology and Neuroscience Johns Hopkins School of Medicine Baltimore Maryland USA

3. Ann & Robert H. Lurie Children's Hospital Chicago Illinois USA

4. Clinical Development, Sarepta Therapeutics, Inc Cambridge Massachusetts USA

5. Clinical Pharmacology, Sarepta Therapeutics, Inc Cambridge Massachusetts USA

6. Pharmacovigilance, Sarepta Therapeutics, Inc Cambridge Massachusetts USA

7. Biostatistics, Sarepta Therapeutics, Inc Cambridge Massachusetts USA

8. Department of Neurology University of California, Los Angeles California USA

Publisher

Wiley

Subject

Physiology (medical),Cellular and Molecular Neuroscience,Clinical Neurology,Physiology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/mus.27347

Reference23 articles.

1. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis

2. Current and emerging treatment strategies for Duchenne muscular dystrophy

3. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

4. Eteplirsen in the treatment of Duchenne muscular dystrophy

5. Sarepta Therapeutics Inc.Exondys® (eteplirsen) injection for intravenous use. US prescribing information.2020.

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