scholarly journals Hemojuvelin is a novel suppressor for Duchenne muscular dystrophy and age‐related muscle wasting

2019 ◽  
Vol 10 (3) ◽  
pp. 557-573 ◽  
Author(s):  
Peng Zhang ◽  
Jian He ◽  
Fei Wang ◽  
Jing Gong ◽  
Lu Wang ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Wasting ◽  
Age Related

Muscle wasting and carbohydrate homeostasis in Duchenne muscular dystrophy

Neurology ◽  
1978 ◽  
Vol 28 (12) ◽  
pp. 1224-1224 ◽  
Author(s):  
M. W. Haymond ◽  
K. E. Strobel ◽  
D. C. DeVivo
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Wasting ◽  
Carbohydrate Homeostasis

scholarly journals The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle

Cells ◽  
2020 ◽  
Vol 9 (12) ◽  
pp. 2657
Author(s):  
Emma Rybalka ◽  
Cara Timpani ◽  
Danielle Debruin ◽  
Ryan Bagaric ◽  
Dean Campelj ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Clinical Data ◽  
Muscle Wasting ◽  
Secondary Outcome ◽  
Disease Course ◽  
Biological Mechanisms ◽  
Inhibitor Development ◽  
Myostatin Inhibition ◽  
Deficient Mice

Myostatin inhibition therapy has held much promise for the treatment of muscle wasting disorders. This is particularly true for the fatal myopathy, Duchenne Muscular Dystrophy (DMD). Following on from promising pre-clinical data in dystrophin-deficient mice and dogs, several clinical trials were initiated in DMD patients using different modality myostatin inhibition therapies. All failed to show modification of disease course as dictated by the primary and secondary outcome measures selected: the myostatin inhibition story, thus far, is a failed clinical story. These trials have recently been extensively reviewed and reasons why pre-clinical data collected in animal models have failed to translate into clinical benefit to patients have been purported. However, the biological mechanisms underlying translational failure need to be examined to ensure future myostatin inhibitor development endeavors do not meet with the same fate. Here, we explore the biology which could explain the failed translation of myostatin inhibitors in the treatment of DMD.

scholarly journals The Failed Clinical Story of Myostatin Inhibitors Against Duchenne Muscular Dystrophy: Exploring the Biology Behind the Battle

2020 ◽  
Author(s):  
Emma Rybalka ◽  
Cara Timpani ◽  
Danielle Debruin ◽  
Ryan Bagaric ◽  
Dean Campelj ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Clinical Data ◽  
Muscle Wasting ◽  
Disease Course ◽  
Biological Mechanisms ◽  
Outcomes Measures ◽  
Myostatin Inhibition ◽  
Secondary Outcomes ◽  
Deficient Mice

Myostatin inhibition therapy has held much promise for the treatment of muscle wasting disorders. This is particularly true for the fatal myopathy, Duchenne Muscular Dystrophy (DMD). Following on from promising pre-clinical data in dystrophin-deficient mice and dogs, several clinical trials were initiated in DMD patients using different modality myostatin inhibition therapies. All failed to show modification of disease course as dictated by the primary and secondary outcomes measures selected: the myostatin inhibition story thus far, is a failed clinical story. These trials have recently been extensively reviewed and reasons why pre-clinical data collected in animal models has failed to translate into clinical benefit to patients has been purported. However, the biological mechanisms underlying translational failure need to be examined to ensure future myostatin inhibitor development endeavors do not meet with the same fate. Here, we explore the biology which could explain the failed translation of myostatin inhibitors in the treatment of DMD.

scholarly journals Special Issue: Pathophysiology and Therapeutic Perspectives in DMD: The Well-Defined Role of the Immune System

Biomedicines ◽  
2021 ◽  
Vol 9 (12) ◽  
pp. 1911
Author(s):  
Andrea Farini
Keyword(s):  
Immune System ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Wasting ◽  
Special Issue ◽  
Wasting Disease ◽  
Muscle Wasting Disease

Duchenne muscular dystrophy (DMD) is the most common, lethal, muscle-wasting disease of childhood [...]

scholarly journals Muscular dystrophy: the route to a cure: Genetic and cell therapies

2008 ◽  
Vol 30 (3) ◽  
pp. 18-21
Author(s):  
Taeyoung Koo ◽  
Takis Athanasopoulos ◽  
George Dickson
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Wasting ◽  
Therapeutic Approaches ◽  
Childhood Diseases ◽  
Cell Therapies ◽  
New Therapies ◽  
Clinical Evaluations ◽  
Dystrophin Protein ◽  
The Way

Duchenne muscular dystrophy (DMD) is one of the most common Xlinked and lifethreatening childhood diseases and affects about 1 in 3000 newborn boys. Lack of dystrophin protein causes severe progressive muscle wasting and death in the second/third decade of life, due to breathing and circulatory complications. Currently, there are no effective medications for DMD, but many differ ent therapeutic approaches are under active development. In the case of genetic and cell therapies, preclinical and clinical evaluations of safety and validity are paving the way towards effective new therapies which could be available routinely for DMD patients in the next 5 years.

scholarly journals Metabogenic and Nutriceutical Approaches to Address Energy Dysregulation and Skeletal Muscle Wasting in Duchenne Muscular Dystrophy

Nutrients ◽  
2015 ◽  
Vol 7 (12) ◽  
pp. 9734-9767 ◽  
Author(s):  
Emma Rybalka ◽  
Cara Timpani ◽  
Christos Stathis ◽  
Alan Hayes ◽  
Matthew Cooke
Keyword(s):  
Skeletal Muscle ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Wasting ◽  
Skeletal Muscle Wasting

scholarly journals Abnormal Calcium Handling in Duchenne Muscular Dystrophy: Mechanisms and Potential Therapies

2021 ◽  
Vol 12 ◽  
Author(s):  
Satvik Mareedu ◽  
Emily D. Million ◽  
Dongsheng Duan ◽  
Gopal J. Babu
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Therapeutic Strategy ◽  
Muscle Wasting ◽  
Premature Death ◽  
Calcium Handling ◽  
Muscle Degeneration ◽  
Wasting Disease ◽  
Fatty Replacement ◽  
Muscle Wasting Disease

Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disease caused by the loss of dystrophin. DMD is associated with muscle degeneration, necrosis, inflammation, fatty replacement, and fibrosis, resulting in muscle weakness, respiratory and cardiac failure, and premature death. There is no curative treatment. Investigations on disease-causing mechanisms offer an opportunity to identify new therapeutic targets to treat DMD. An abnormal elevation of the intracellular calcium (Cai2+) concentration in the dystrophin-deficient muscle is a major secondary event, which contributes to disease progression in DMD. Emerging studies have suggested that targeting Ca2+-handling proteins and/or mechanisms could be a promising therapeutic strategy for DMD. Here, we provide an updated overview of the mechanistic roles the sarcolemma, sarcoplasmic/endoplasmic reticulum, and mitochondria play in the abnormal and sustained elevation of Cai2+ levels and their involvement in DMD pathogenesis. We also discuss current approaches aimed at restoring Ca2+ homeostasis as potential therapies for DMD.

scholarly journals Age-Related Longitudinal Changes in Metabolic Energy Expenditure during Walking in Boys with Duchenne Muscular Dystrophy

PLoS ONE ◽  
2014 ◽  
Vol 9 (12) ◽  
pp. e115200 ◽  
Author(s):  
Merel-Anne Brehm ◽  
Jiska C. E. Kempen ◽  
Anneke J. van der Kooi ◽  
Imelda J. M. de Groot ◽  
Janneke C. van den Bergen ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Energy Expenditure ◽  
Metabolic Energy ◽  
Longitudinal Changes ◽  
Age Related ◽  
Metabolic Energy Expenditure
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