The CINRG Becker Natural History Study: Baseline characteristics-Reference-Cited by-同舟云学术

The CINRG Becker Natural History Study: Baseline characteristics

Published:2020-07-24 Issue:3 Volume:62 Page:369-376
ISSN:0148-639X
Container-title:Muscle & Nerve
language:en
Short-container-title:Muscle & Nerve

Author:

Clemens Paula R.¹^ORCID,Niizawa Gabriela²,Feng Jia³,Florence Julaine⁴,DʼAlessandro Andrea Smith²,Morgenroth Lauren P.⁵,Gorni Ksenija⁶,Guglieri Michela⁷,Connolly Anne⁴,Wicklund Matthew⁸,Bertorini Tulio⁹,Mah Jean K.¹⁰,Thangarajh Mathula³,Smith Edward¹¹,Kuntz Nancy¹²,McDonald Craig M.¹³,Henricson Erik K.¹³^ORCID,Upadhyayula Saila¹⁴,Byrne Barry¹⁵,Manousakis Georgios¹⁶,Harper Amy¹⁷,Bravver Elena¹⁷,Iannaccone Susan¹⁸,Spurney Christopher³,Cnaan Avital³,Gordish‐Dressman Heather³,

Affiliation:

1. Department of Veterans Affairs Medical Center and University of Pittsburgh School of Medicine Pittsburgh Pennsylvania USA

2. University of Pittsburgh School of Medicine Pittsburgh Pennsylvania USA

3. Childrenʼs National Medical Center Washington, DC USA

4. Washington University St. Louis Missouri USA

5. TRiNDS Pittsburgh Pennsylvania USA

6. Centro Clinico Nemo Hospital Milan Italy

7. Newcastle University Newcastle Upon Tyne UK

8. Hershey Medical Center Hershey Pennsylvania USA

9. University of Tennessee Memphis Tennessee USA

10. Alberta Childrenʼs Hospital Calgary Alberta Canada

11. Duke University Medical Center Durham North Carolina USA

12. Lurie Childrenʼs Hospital Chicago Illinois USA

13. University of California Davis Sacramento California USA

14. Childrenʼs Healthcare of Atlanta Atlanta Georgia USA

15. University of Florida Gainesville Florida USA

16. University of Minnesota Minneapolis Minnesota USA

17. Carolinas Medical Center Charlotte North Carolina USA

18. University of Texas Southwestern Medical Center Dallas Texas USA

Funder

Muscular Dystrophy Association

National Institute of Arthritis and Musculoskeletal and Skin Diseases

Publisher

Wiley

Subject

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

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/mus.27011

Reference27 articles.

1. Exploring the molecular basis for variability among patients with Becker muscular dystrophy: dystrophin gene and protein studies;Beggs AH;Am J Hum Genet,1991

2. Characterization of Dystrophin in Muscle-Biopsy Specimens from Patients with Duchenne's or Becker's Muscular Dystrophy

3. The clinical, genetic and dystrophin characteristics of Becker muscular dystrophy

4. Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

5. Eteplirsen for the treatment of Duchenne muscular dystrophy

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1. Findings from the Longitudinal CINRG Becker Natural History Study;Journal of Neuromuscular Diseases;2024-01-02

2. Natural history of Becker muscular dystrophy: a multicenter study of 225 patients;Annals of Clinical and Translational Neurology;2023-10-26

3. CRISPR-Cas9 homology-independent targeted integration of exons 1–19 restores full-length dystrophin in mice;Molecular Therapy - Methods & Clinical Development;2023-09

4. An update on Becker muscular dystrophy;Current Opinion in Neurology;2023-08-21

5. Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice;iScience;2023-07