Literature DB >> 25037084

Duchenne and Becker muscular dystrophies.

Kevin M Flanigan1.   

Abstract

The dystrophinopathies Duchenne and Becker muscular dystrophies (DMD and BMD) represent the most common inherited disorders of muscle. Improvements in cardiac care, attention to respiratory function, and judicious use of spinal correction surgery have led to increased survival in the DMD population. Meanwhile, advances in molecular therapeutics have led to promising therapies that are in or are entering clinical trials. An understanding of the dystrophinopathies, and recent advances in their molecular diagnosis and treatment, is of benefit to practicing neurologists.
Copyright © 2014 Elsevier Inc. All rights reserved.

Entities:  

Keywords:  Becker muscular dystrophy; Duchenne muscular dystrophy; Dystrophin; Exon skipping

Mesh:

Substances:

Year:  2014        PMID: 25037084     DOI: 10.1016/j.ncl.2014.05.002

Source DB:  PubMed          Journal:  Neurol Clin        ISSN: 0733-8619            Impact factor:   3.806


  90 in total

1.  Sunitinib promotes myogenic regeneration and mitigates disease progression in the mdx mouse model of Duchenne muscular dystrophy.

Authors:  Tatiana M Fontelonga; Brennan Jordan; Andreia M Nunes; Pamela Barraza-Flores; Nicholas Bolden; Ryan D Wuebbles; Lesley Mathews Griner; Xin Hu; Marc Ferrer; Juan Marugan; Noel Southall; Dean J Burkin
Journal:  Hum Mol Genet       Date:  2019-07-01       Impact factor: 6.150

2.  Factors affecting the health-related quality of life of caregivers of patients with muscular dystrophy.

Authors:  Yi-Jing Lue; Shun-Sheng Chen; Yen-Mou Lu
Journal:  J Neurol       Date:  2018-04-26       Impact factor: 4.849

3.  Psychosocial Needs and Facilitators of Mothers Caring for Children with Duchenne/Becker Muscular Dystrophy.

Authors:  Holly L Peay; Bettina Meiser; Kathleen Kinnett; Aad Tibben
Journal:  J Genet Couns       Date:  2017-08-12       Impact factor: 2.537

4.  [Molecular pathogenesis of Duchenne muscular dystrophy-related fibrosis].

Authors:  K Ohlendieck; D Swandulla
Journal:  Pathologe       Date:  2017-02       Impact factor: 1.011

Review 5.  Dystrophin-deficient large animal models: translational research and exon skipping.

Authors:  Xinran Yu; Bo Bao; Yusuke Echigoya; Toshifumi Yokota
Journal:  Am J Transl Res       Date:  2015-08-15       Impact factor: 4.060

Review 6.  Muscle MRI in pediatrics: clinical, pathological and genetic correlation.

Authors:  Claudia P Cejas; Maria M Serra; David F Gonzalez Galvez; Eliana A Cavassa; Ana L Taratuto; Gabriel A Vazquez; Mario E L Massaro; Angeles V Schteinschneider
Journal:  Pediatr Radiol       Date:  2017-01-19

Review 7.  A human-on-a-chip approach to tackling rare diseases.

Authors:  Camilly P Pires de Mello; John Rumsey; Victoria Slaughter; James J Hickman
Journal:  Drug Discov Today       Date:  2019-08-11       Impact factor: 7.851

8.  Bone Health and Endocrine Care of Boys with Duchenne Muscular Dystrophy: Data from the MD STARnet.

Authors:  David R Weber; Shiny Thomas; Stephen W Erickson; Deborah Fox; Joyce Oleszek; Shree Pandya; Yedatore Venkatesh; Christina Westfield; Emma Ciafaloni
Journal:  J Neuromuscul Dis       Date:  2018

9.  Prioritizing Parental Worry Associated with Duchenne Muscular Dystrophy Using Best-Worst Scaling.

Authors:  Holly Landrum Peay; I L Hollin; J F P Bridges
Journal:  J Genet Couns       Date:  2015-08-21       Impact factor: 2.537

Review 10.  Hydrogen sulfide pathway and skeletal muscle: an introductory review.

Authors:  Valentina Vellecco; Chiara Armogida; Mariarosaria Bucci
Journal:  Br J Pharmacol       Date:  2018-06-15       Impact factor: 8.739

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