Literature DB >> 33663533

Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?

Stephanie Kourakis1, Cara A Timpani1,2, Dean G Campelj1,2, Patricia Hafner3, Nuri Gueven4, Dirk Fischer3, Emma Rybalka5,6.   

Abstract

BACKGROUND: Pharmacological corticosteroid therapy is the standard of care in Duchenne Muscular Dystrophy (DMD) that aims to control symptoms and slow disease progression through potent anti-inflammatory action. However, a major concern is the significant adverse effects associated with long term-use. MAIN: This review discusses the pros and cons of standard of care treatment for DMD and compares it to novel data generated with the new-wave dissociative corticosteroid, vamorolone. The current status of experimental anti-inflammatory pharmaceuticals is also reviewed, with insights regarding alternative drugs that could provide therapeutic advantage.
CONCLUSIONS: Although novel dissociative steroids may be superior substitutes to corticosteroids, other potential therapeutics should be explored. Repurposing or developing novel pharmacological therapies capable of addressing the many pathogenic features of DMD in addition to anti-inflammation could elicit greater therapeutic advantages.

Entities:  

Keywords:  Anti-inflammation; Anti-inflammatory drugs; Corticosteroids; Duchenne muscular dystrophy; Standard of care

Mesh:

Substances:

Year:  2021        PMID: 33663533      PMCID: PMC7934375          DOI: 10.1186/s13023-021-01758-9

Source DB:  PubMed          Journal:  Orphanet J Rare Dis        ISSN: 1750-1172            Impact factor:   4.123


  129 in total

1.  Prednisolone in Duchenne muscular dystrophy with imminent loss of ambulation.

Authors:  Sunil Pradhan; Debabrata Ghosh; Niraj Kumar Srivastava; Ashok Kumar; Balraj Mittal; Chandra Mani Pandey; Uttam Singh
Journal:  J Neurol       Date:  2006-06-19       Impact factor: 4.849

2.  Prednisone in Duchenne dystrophy. A randomized, controlled trial defining the time course and dose response. Clinical Investigation of Duchenne Dystrophy Group.

Authors:  R C Griggs; R T Moxley; J R Mendell; G M Fenichel; M H Brooke; A Pestronk; J P Miller
Journal:  Arch Neurol       Date:  1991-04

3.  Associations between timing of corticosteroid treatment initiation and clinical outcomes in Duchenne muscular dystrophy.

Authors:  Sunkyung Kim; Yong Zhu; Paul A Romitti; Deborah J Fox; Daniel W Sheehan; Rodolfo Valdez; Dennis Matthews; Brent J Barber
Journal:  Neuromuscul Disord       Date:  2017-06-05       Impact factor: 4.296

Review 4.  Activation of Nrf2-antioxidant signaling attenuates NFkappaB-inflammatory response and elicits apoptosis.

Authors:  Wenge Li; Tin Oo Khor; Changjiang Xu; Guoxiang Shen; Woo-Sik Jeong; Siwang Yu; Ah-Ng Kong
Journal:  Biochem Pharmacol       Date:  2008-07-23       Impact factor: 5.858

5.  Low-dose prednisolone treatment in Duchenne and Becker muscular dystrophy.

Authors:  E Bäckman; K G Henriksson
Journal:  Neuromuscul Disord       Date:  1995-05       Impact factor: 4.296

6.  Glucocorticoid Receptor-mediated transactivation is hampered by Striatin-3, a novel interaction partner of the receptor.

Authors:  Ioanna Petta; Nadia Bougarne; Jolien Vandewalle; Lien Dejager; Sofie Vandevyver; Marlies Ballegeer; Sofie Desmet; Jonathan Thommis; Lode De Cauwer; Sam Lievens; Claude Libert; Jan Tavernier; Karolien De Bosscher
Journal:  Sci Rep       Date:  2017-08-21       Impact factor: 4.379

Review 7.  Glucocorticosteroid therapy in inflammatory bowel diseases: From clinical practice to molecular biology.

Authors:  Karen Dubois-Camacho; Payton A Ottum; Daniel Franco-Muñoz; Marjorie De la Fuente; Alejandro Torres-Riquelme; David Díaz-Jiménez; Mauricio Olivares-Morales; Gonzalo Astudillo; Rodrigo Quera; Marcela A Hermoso
Journal:  World J Gastroenterol       Date:  2017-09-28       Impact factor: 5.742

Review 8.  Glucocorticoids-All-Rounders Tackling the Versatile Players of the Immune System.

Authors:  Cindy Strehl; Lisa Ehlers; Timo Gaber; Frank Buttgereit
Journal:  Front Immunol       Date:  2019-07-24       Impact factor: 7.561

9.  Phase 1 Study of a Sulforaphane-Containing Broccoli Sprout Homogenate for Sickle Cell Disease.

Authors:  Jennifer F Doss; Jude C Jonassaint; Melanie E Garrett; Allison E Ashley-Koch; Marilyn J Telen; Jen-Tsan Chi
Journal:  PLoS One       Date:  2016-04-12       Impact factor: 3.240

10.  Prednisone and Deflazacort in Duchenne Muscular Dystrophy: Do They Play a Different Role in Child Behavior and Perceived Quality of Life?

Authors:  Susan Sienko; Cathleen Buckon; Eileen Fowler; Anita Bagley; Loretta Staudt; Mitell Sison-Williamson; Kathy Zebracki; Craig M McDonald; Michael Sussman
Journal:  PLoS Curr       Date:  2016-06-17
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  7 in total

1.  The association between cardiac involvement and long-term clinical outcomes in patients with Duchenne muscular dystrophy.

Authors:  Jung-Joon Cha; In-Soo Kim; Jong-Youn Kim; Eui-Young Choi; Pil-Ki Min; Young Won Yoon; Byoung Kwon Lee; Bum-Kee Hong; Hyuck Moon Kwon; Han Eol Cho; Won Ah Choi; Seong-Woong Kang; Se-Joong Rim
Journal:  ESC Heart Fail       Date:  2022-05-17

2.  Myxomavirus Serp-1 Protein Ameliorates Inflammation in a Mouse Model of Duchenne Muscular Dystrophy.

Authors:  Alexander B Andre; Liqiang Zhang; Jalen D Nix; Nora Elmadbouly; Alexandra R Lucas; Jeanne Wilson-Rawls; Alan Rawls
Journal:  Biomedicines       Date:  2022-05-17

3.  β-Glucans as Dietary Supplement to Improve Locomotion and Mitochondrial Respiration in a Model of Duchenne Muscular Dystrophy.

Authors:  Letizia Brogi; Maria Marchese; Alessandro Cellerino; Rosario Licitra; Valentina Naef; Serena Mero; Carlo Bibbiani; Baldassare Fronte
Journal:  Nutrients       Date:  2021-05-12       Impact factor: 5.717

4.  β-Dystroglycan Restoration and Pathology Progression in the Dystrophic mdx Mouse: Outcome and Implication of a Clinically Oriented Study with a Novel Oral Dasatinib Formulation.

Authors:  Paola Mantuano; Brigida Boccanegra; Elena Conte; Michela De Bellis; Santa Cirmi; Francesca Sanarica; Ornella Cappellari; Ilaria Arduino; Annalisa Cutrignelli; Angela Assunta Lopedota; Antonietta Mele; Nunzio Denora; Annamaria De Luca
Journal:  Biomolecules       Date:  2021-11-22

5.  Sphingosine Phosphate Lyase Is Upregulated in Duchenne Muscular Dystrophy, and Its Inhibition Early in Life Attenuates Inflammation and Dystrophy in Mdx Mice.

Authors:  Anabel S De la Garza-Rodea; Steven A Moore; Jesus Zamora-Pineda; Eric P Hoffman; Karishma Mistry; Ashok Kumar; Jonathan B Strober; Piming Zhao; Jung H Suh; Julie D Saba
Journal:  Int J Mol Sci       Date:  2022-07-08       Impact factor: 6.208

6.  Exploring the Therapeutic Potential of Ectoine in Duchenne Muscular Dystrophy: Comparison with Taurine, a Supplement with Known Beneficial Effects in the mdx Mouse.

Authors:  Caroline Merckx; Jana Zschüntzsch; Stefanie Meyer; Robrecht Raedt; Hanne Verschuere; Jens Schmidt; Boel De Paepe; Jan L De Bleecker
Journal:  Int J Mol Sci       Date:  2022-08-24       Impact factor: 6.208

7.  miR-378 affects metabolic disturbances in the mdx model of Duchenne muscular dystrophy.

Authors:  Paulina Podkalicka; Olga Mucha; Katarzyna Kaziród; Krzysztof Szade; Jacek Stępniewski; Liudmyla Ivanishchuk; Hirofumi Hirao; Ewelina Pośpiech; Alicja Józkowicz; Jerzy W Kupiec-Weglinski; Józef Dulak; Agnieszka Łoboda
Journal:  Sci Rep       Date:  2022-03-10       Impact factor: 4.996

  7 in total

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