Literature DB >> 23137739

Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy.

Eric P Hoffman1, Erica Reeves, Jesse Damsker, Kanneboyina Nagaraju, John M McCall, Edward M Connor, Kate Bushby.   

Abstract

Although prednisone has never been formally approved for use in Duchenne muscular dystrophy (DMD) by regulatory agencies, its efficacy has been confirmed in trials dating from the 1980s. There is a strong need for optimization of both specific type of glucocorticoid (eg, prednisone, vs deflazacort or others) and the dosing regimen. Ideally an optimized regimen would maximize efficacy while minimizing side-effect profiles. A new trial, FOR-DMD, aims to address this gap in knowledge. In parallel, there has been progress in the area of "dissociative steroids," drugs that are able to better separate efficacy and side effects, providing a broader therapeutic window.
Copyright © 2012 Elsevier Inc. All rights reserved.

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Year:  2012        PMID： 23137739      PMCID： PMC3606917          DOI： 10.1016/j.pmr.2012.08.003

Source DB:  PubMed          Journal:  Phys Med Rehabil Clin N Am        ISSN： 1047-9651            Impact factor:   1.784


  35 in total

1.  Balancing muscle hypertrophy and atrophy.

Authors:  Eric P Hoffman; Gustavo A Nader
Journal:  Nat Med       Date:  2004-06       Impact factor: 53.440

Review 2.  Glucocorticoid-induced myopathy.

Authors:  Rosa Maria Rodrigues Pereira; Jozélio Freire de Carvalho
Journal:  Joint Bone Spine       Date:  2010-05-14       Impact factor: 4.929

3.  The mechanism of cytoprotective action of lazaroids I: Inhibition of reactive oxygen species formation and lethal cell injury during periods of energy depletion.

Authors:  B M Taylor; W E Fleming; C W Benjamin; Y Wu; W R Mathews; F F Sun
Journal:  J Pharmacol Exp Ther       Date:  1996-03       Impact factor: 4.030

4.  Administration of methylprednisolone for 24 or 48 hours or tirilazad mesylate for 48 hours in the treatment of acute spinal cord injury. Results of the Third National Acute Spinal Cord Injury Randomized Controlled Trial. National Acute Spinal Cord Injury Study.

Authors:  M B Bracken; M J Shepard; T R Holford; L Leo-Summers; E F Aldrich; M Fazl; M Fehlings; D L Herr; P W Hitchon; L F Marshall; R P Nockels; V Pascale; P L Perot; J Piepmeier; V K Sonntag; F Wagner; J E Wilberger; H R Winn; W Young
Journal:  JAMA       Date:  1997-05-28       Impact factor: 56.272

5.  Long-Term Steroid Therapy in Duchenne Muscular Dystrophy-Positive Results versus Side Effects.

Authors:  U Schara; W Mortier
Journal:  J Clin Neuromuscul Dis       Date:  2001-06

6.  Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals.

Authors:  M Koenig; E P Hoffman; C J Bertelson; A P Monaco; C Feener; L M Kunkel
Journal:  Cell       Date:  1987-07-31       Impact factor: 41.582

Review 7.  Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care.

Authors:  Katharine Bushby; Richard Finkel; David J Birnkrant; Laura E Case; Paula R Clemens; Linda Cripe; Ajay Kaul; Kathi Kinnett; Craig McDonald; Shree Pandya; James Poysky; Frederic Shapiro; Jean Tomezsko; Carolyn Constantin
Journal:  Lancet Neurol       Date:  2009-11-27       Impact factor: 44.182

8.  Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy.

Authors:  Larry W Markham; Kathi Kinnett; Brenda L Wong; D Woodrow Benson; Linda H Cripe
Journal:  Neuromuscul Disord       Date:  2008-04-23       Impact factor: 4.296

9.  Steroids in Duchenne muscular dystrophy--deflazacort trial.

Authors:  L E Mesa; A L Dubrovsky; J Corderi; P Marco; D Flores
Journal:  Neuromuscul Disord       Date:  1991       Impact factor: 4.296

Review 10.  The role of corticosteroids in muscular dystrophy: a critical appraisal.

Authors:  Corrado Angelini
Journal:  Muscle Nerve       Date:  2007-10       Impact factor: 3.217
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  26 in total

1.  Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study.

Authors:  Luca Bello; Heather Gordish-Dressman; Lauren P Morgenroth; Erik K Henricson; Tina Duong; Eric P Hoffman; Avital Cnaan; Craig M McDonald
Journal:  Neurology       Date:  2015-08-26       Impact factor: 9.910

2.  Two-Year Longitudinal Changes in Lower Limb Strength and Its Relation to Loss in Function in a Large Cohort of Patients With Duchenne Muscular Dystrophy.

Authors:  Abhinandan Batra; Ann Harrington; Donovan J Lott; Rebecca Willcocks; Claudia R Senesac; William McGehee; Dandan Xu; Sunita Mathur; Michael J Daniels; William D Rooney; Sean C Forbes; William Triplett; Jasjit K Deol; Ishu Arpan; Roxanne Bendixen; Richard Finkel; Erika Finanger; Gihan Tennekoon; Barry Byrne; Barry Russman; H Lee Sweeney; Glenn Walter; Krista Vandenborne
Journal:  Am J Phys Med Rehabil       Date:  2018-10       Impact factor: 2.159

3.  Contemporary cardiac issues in Duchenne muscular dystrophy. Working Group of the National Heart, Lung, and Blood Institute in collaboration with Parent Project Muscular Dystrophy.

Authors:  Elizabeth M McNally; Jonathan R Kaltman; D Woodrow Benson; Charles E Canter; Linda H Cripe; Dongsheng Duan; Jonathan D Finder; William J Groh; Eric P Hoffman; Daniel P Judge; Naomi Kertesz; Kathi Kinnett; Roxanne Kirsch; Joseph M Metzger; Gail D Pearson; Jill A Rafael-Fortney; Subha V Raman; Christopher F Spurney; Shari L Targum; Kathryn R Wagner; Larry W Markham
Journal:  Circulation       Date:  2015-05-05       Impact factor: 29.690

Review 4.  Treatment of dystrophin cardiomyopathies.

Authors:  Josef Finsterer; Linda Cripe
Journal:  Nat Rev Cardiol       Date:  2014-01-14       Impact factor: 32.419

5.  VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid.

Authors:  Erica K M Reeves; Eric P Hoffman; Kanneboyina Nagaraju; Jesse M Damsker; John M McCall
Journal:  Bioorg Med Chem       Date:  2013-02-18       Impact factor: 3.641

6.  Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment.

Authors:  Xu Liu; Yashuo Wang; Jennifer S Gutierrez; Jesse M Damsker; Kanneboyina Nagaraju; Eric P Hoffman; Eric A Ortlund
Journal:  Proc Natl Acad Sci U S A       Date:  2020-09-11       Impact factor: 11.205

7.  Histologic muscular history in steroid-treated and untreated patients with Duchenne dystrophy.

Authors:  Lorenzo Peverelli; Silvia Testolin; Luisa Villa; Adele D'Amico; Stefania Petrini; Chiara Favero; Francesca Magri; Lucia Morandi; Marina Mora; Tiziana Mongini; Enrico Bertini; Monica Sciacco; Giacomo P Comi; Maurizio Moggio
Journal:  Neurology       Date:  2015-10-23       Impact factor: 9.910

8.  Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy.

Authors:  Yetrib Hathout; Edward Brody; Paula R Clemens; Linda Cripe; Robert Kirk DeLisle; Pat Furlong; Heather Gordish-Dressman; Lauren Hache; Erik Henricson; Eric P Hoffman; Yvonne Monique Kobayashi; Angela Lorts; Jean K Mah; Craig McDonald; Bob Mehler; Sally Nelson; Malti Nikrad; Britta Singer; Fintan Steele; David Sterling; H Lee Sweeney; Steve Williams; Larry Gold
Journal:  Proc Natl Acad Sci U S A       Date:  2015-05-26       Impact factor: 11.205

9.  The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations.

Authors:  Catherine L Bladen; David Salgado; Soledad Monges; Maria E Foncuberta; Kyriaki Kekou; Konstantina Kosma; Hugh Dawkins; Leanne Lamont; Anna J Roy; Teodora Chamova; Velina Guergueltcheva; Sophelia Chan; Lawrence Korngut; Craig Campbell; Yi Dai; Jen Wang; Nina Barišić; Petr Brabec; Jaana Lahdetie; Maggie C Walter; Olivia Schreiber-Katz; Veronika Karcagi; Marta Garami; Venkatarman Viswanathan; Farhad Bayat; Filippo Buccella; En Kimura; Zaïda Koeks; Janneke C van den Bergen; Miriam Rodrigues; Richard Roxburgh; Anna Lusakowska; Anna Kostera-Pruszczyk; Janusz Zimowski; Rosário Santos; Elena Neagu; Svetlana Artemieva; Vedrana Milic Rasic; Dina Vojinovic; Manuel Posada; Clemens Bloetzer; Pierre-Yves Jeannet; Franziska Joncourt; Jordi Díaz-Manera; Eduard Gallardo; A Ayşe Karaduman; Haluk Topaloğlu; Rasha El Sherif; Angela Stringer; Andriy V Shatillo; Ann S Martin; Holly L Peay; Matthew I Bellgard; Jan Kirschner; Kevin M Flanigan; Volker Straub; Kate Bushby; Jan Verschuuren; Annemieke Aartsma-Rus; Christophe Béroud; Hanns Lochmüller
Journal:  Hum Mutat       Date:  2015-03-17       Impact factor: 4.878

10.  Prednisolone improves walking in Japanese Duchenne muscular dystrophy patients.

Authors:  Fumi Takeuchi; Naohiro Yonemoto; Harumasa Nakamura; Reiko Shimizu; Hirofumi Komaki; Madoka Mori-Yoshimura; Yukiko K Hayashi; Ichizo Nishino; Mitsuru Kawai; En Kimura; Shin'ichi Takeda
Journal:  J Neurol       Date:  2013-09-22       Impact factor: 4.849
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