Steroid Therapy and Cardiac Function in Duchenne Muscular Dystrophy

2005 ◽  
Vol 26 (6) ◽  
pp. 768-771 ◽  
Author(s):  
L.W. Markham ◽  
R.L. Spicer ◽  
P.R. Khoury ◽  
B.L. Wong ◽  
K.D. Mathews ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Cardiac Function ◽  
Steroid Therapy

DUCHENNE MUSCULAR DYSTROPHY AND STEROID THERAPY: EVALUATION OF PATIENTS' MOTOR PERFORMANCE

2006 ◽  
Vol 37 (S 1) ◽  
Author(s):  
SL Parreira ◽  
MBD Resende ◽  
R Cardoso Alves ◽  
MDC Peduto ◽  
MS Carvalho ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Steroid Therapy ◽  
Motor Performance ◽  
Therapy Evaluation

T.P.49 Low-dose steroid therapy for cardiomyopathy in Duchenne Muscular Dystrophy patients

2012 ◽  
Vol 22 (9-10) ◽  
pp. 866
Author(s):  
K. Ishigaki ◽  
T. Murakami ◽  
T. Saito ◽  
T. Sato ◽  
S. Kajino ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Steroid Therapy ◽  
Low Dose

Vitamin D in the prevention and treatment of comorbid conditions in Duchenne muscular dystrophy

2021 ◽  
Vol 2 (1) ◽  
pp. 38-50
Author(s):  
Tatiana A. Gremiakova ◽  
Vasiliy M. Souslov ◽  
Gulzhan E. Sakbaeva ◽  
Andrey A. Stepanov
Keyword(s):  
Vitamin D ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Steroid Therapy ◽  
Motor Development ◽  
Smooth Muscles ◽  
Comorbid Conditions ◽  
Increased Risk ◽  
Positive Effect ◽  
Tubular Bones

Duchenne muscular dystrophy (DMD) is an X-linked recessive degenerative neuromuscular disorder due to a deficiency of dystrophin protein. This protein is most common in skeletal and cardiac muscles, to a lesser extent in smooth muscles and the brain. With DMD, progressive damage and muscle degeneration, a delay in motor development, and respiratory cardiac disorders are progressing. Patients with DMD have an increased risk of developing osteoporosis, fractures of the tubular bones and vertebrae, and neurocognitive impairment. Vitamin D is recommended prophylactically for DMD since many studies have shown its deficiency. The purpose of this work is to consolidate the literature data on the vitamin D deficiency in DMD patients and its effects on the development of concurrent comorbid conditions of the musculoskeletal, endocrine, and nervous systems. The authors discuss data concerning the appropriate level of vitamin D throughout the life span of DMD has a positive effect on the course of the disease patients’ quality of life ends. Primary clinical outcomes of vitamin D normalization include prevention of the development of osteoporosis (especially after the start of steroid therapy), fractures of the tubular bones and vertebrae, prolonged ability to walk, more effective treatment with bisphosphonates, including a decrease in the number of complications during initial use and lower jaw necrosis, positive effect on the expression of autistic spectrum symptoms. For patients with long-term steroid therapy, metabolic and liver disorders, calcidiol could be used, allowing quick deficiency compensation instead of standard vitamin D preparations.

Ifetroban Reduces Coronary Artery Dysfunction in a Mouse Model of Duchenne Muscular Dystrophy

2021 ◽  
Author(s):  
Ray Mitchell ◽  
Norman E Frederick ◽  
Emily R Holzman ◽  
Francesca Agobe ◽  
Heather C M Allaway ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Cardiac Function ◽  
Dilated Cardiomyopathy ◽  
Coronary Arteries ◽  
Oral Treatment ◽  
Mdx Mice ◽  
Muscarinic Agonist ◽  
Therapeutic Implications

Dilated cardiomyopathy contributes to morbidity and mortality in Duchenne Muscular Dystrophy (DMD), an inheritable muscle wasting disease caused by a mutation in the dystrophin gene. Preclinical studies in mouse models of muscular dystrophy have demonstrated reduced cardiomyopathy and improved cardiac function following oral treatment with the potent and selective thromboxane A2/prostanoid receptor (TPr) antagonist, ifetroban. Further, a phase 2 clinical trial (NCT03340675, Cumberland Pharmaceutical) is currently recruiting subjects to determine if ifetroban can improve cardiac function in patients with DMD. Although TPr is a promising therapeutic target for the treatment of dilated cardiomyopathy in DMD, little is known about TPr function in coronary arteries that perfuse blood through the cardiac tissue. In the current study, isolated coronary arteries from young (~3-5 months) and aged (~9-12 months) mdx mice, a widely used mouse model of DMD, and age-matched controls were examined using wire myography. Vasoconstriction to increasing concentrations of TPr agonist U-46619(U4) was enhanced in young mdx mice versus controls. Additionally, young mdx mice displayed a significant attenuation in endothelial cell-mediated vasodilation to increasing concentrations of the muscarinic agonist acetylcholine (ACh). Since TPr activation was enhanced in young mdx mice, U4-mediated vasoconstriction was measured in the absence and presence of ifetroban. Ifetroban reduced U4-mediated vasoconstriction in young mdx and both aged mdx and control mice. Overall, our data demonstrate enhanced coronary arterial vasoconstriction to TPr activation in young mdx mice, a phenotype that could be reversed with ifetroban. These data could have important therapeutic implications for improving cardiovascular function in DMD.

Steroid therapy in duchenne muscular dystrophy (DMD)

1992 ◽  
Vol 8 (5) ◽  
pp. 353
Author(s):  
Alberto L. Dubrovsky ◽  
Lilia Mesa ◽  
José Corderi ◽  
Patricia Marco ◽  
Daniel Flores
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Steroid Therapy

scholarly journals Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy

PLoS ONE ◽  
2018 ◽  
Vol 13 (6) ◽  
pp. e0198897 ◽  
Author(s):  
Alison M. Blain ◽  
Elizabeth Greally ◽  
Graham McClorey ◽  
Raquel Manzano ◽  
Corinne A. Betts ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Cardiac Function ◽  
Mouse Models ◽  
Exon Skipping ◽  
Mdx Mouse

Trends of steroid therapy for Duchenne muscular dystrophy in Japan

2015 ◽  
Vol 25 ◽  
pp. S199
Author(s):  
F. Takeuchi ◽  
H. Komaki ◽  
H. Nakamura ◽  
N. Yonemoto ◽  
K. Kashiwabara ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Steroid Therapy

scholarly journals Reversal of cardiac and skeletal manifestations of Duchenne muscular dystrophy by cardiosphere-derived cells and their exosomes in mdx dystrophic mice and in human Duchenne cardiomyocytes

2017 ◽  
Author(s):  
Mark A. Aminzadeh ◽  
Russell G. Rogers ◽  
Kenneth Gouin ◽  
Mario Fournier ◽  
Rachel E. Tobin ◽  
...  
Keyword(s):  
Skeletal Muscle ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Mouse Model ◽  
Cardiac Function ◽  
Premature Death ◽  
Next Generation ◽  
Skeletal Myopathy ◽  
Genetic Deficiency ◽  
Dystrophic Mice

Genetic deficiency of dystrophin leads to disability and premature death in Duchenne muscular dystrophy, affecting the heart as well as skeletal muscle. Here we report that cardiosphere-derived cells (CDCs), which are being tested clinically for the treatment of Duchenne cardiomyopathy, improve cardiac and skeletal myopathy in the mdx mouse model of DMD and in human Duchenne cardiomyocytes. Injection of CDCs into the hearts of mdx mice augments cardiac function, ambulatory capacity and survival. Exosomes secreted by human CDCs reproduce the benefits of CDCs in mdx mice and in human Duchenne cardiomyocytes. The findings further motivate the testing of CDCs in Duchenne patients, while identifying exosomes as next-generation therapeutic candidates.

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