Tuesday, 12 July 2016

PPMD Awards The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne - PPMD Community

PPMD Awards The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne - PPMD Community: "Parent Project Muscular Dystrophy (PPMD) announced today plans to award Dr. Denis Guttridge of The Ohio State University with a $48,000 grant for his work in cardiac issues in Duchenne. Duchenne affects muscles, and since the heart is a muscle too, cardiac problems remain a major concern for patients. PPMD has spent the last several years supporting efforts to improve cardiac care and believe in the promising work Dr. Guttridge and his team at Ohio State are doing.
 
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births. Almost all Duchenne patients develop heart problems. In order to treat the disease, new drugs will have to work by fixing both skeletal and heart muscles. In contrast to Duchenne skeletal muscles, very little is known about a dystrophic heart."



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PPMD Awards The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne - PPMD Community

PPMD Awards The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne - PPMD Community: "Parent Project Muscular Dystrophy (PPMD) announced today plans to award Dr. Denis Guttridge of The Ohio State University with a $48,000 grant for his work in cardiac issues in Duchenne. Duchenne affects muscles, and since the heart is a muscle too, cardiac problems remain a major concern for patients. PPMD has spent the last several years supporting efforts to improve cardiac care and believe in the promising work Dr. Guttridge and his team at Ohio State are doing.
 
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births. Almost all Duchenne patients develop heart problems. In order to treat the disease, new drugs will have to work by fixing both skeletal and heart muscles. In contrast to Duchenne skeletal muscles, very little is known about a dystrophic heart."



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LIF-treated muscle stem cells show promise in treatment of muscular dystrophy

LIF-treated muscle stem cells show promise in treatment of muscular dystrophy: "Satellite cells are stem cells found in skeletal muscles. While transplantation of such muscle stem cells can be a potent therapy for degenerative muscle diseases such as Duchenne muscular dystrophy, these cells tend to lose their transplantation efficiency when cultured in vitro. In a study in the current issue of the Journal of Neuromuscular Diseases, researchers treated these stem cells with leukemia inhibitory factor (LIF), which effectively maintained the undifferentiated state of the satellite cells and enhanced their transplantation efficiency.

To have enough cells for transplantation, they must be grown in vitro and prevented from differentiating before transplantation. Several growth factors, cytokines, and chemicals have been used in muscle stem cell cultures, but the optimal culture conditions required to maintain the undifferentiated state, inhibit differentiation, and enhance eventual transplantation efficiency have not yet been established."



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Tuesday, 28 June 2016

New Duchenne muscular dystrophy treatment 'ready in five years' - BBC News

New Duchenne muscular dystrophy treatment 'ready in five years' - BBC News: "A new treatment for a rare muscle wasting disease could be available within five years, an Oxford University researcher has said.
Duchenne muscular dystrophy (DMD) affects about 2,500 boys and men in the UK, leaving them unable to move.
Professor Kay Davies said trials of a drug to increase levels of the protein utrophin, to maintain the muscles, would start later this year.
She told Oxfordshire Science Festival it could help sufferers worldwide.
"Duchenne is horrible for these boys. They normally get diagnosed at the age of four of five, and they suffer from a progressive muscle wastage which leaves them in a wheelchair by the age of 12," she said.
"Somewhere in the next five years, we will be able to do something for these boys, to stop them from going into a wheelchair, and perhaps prolong their life and improve their quality of life."
"



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Saturday, 18 June 2016

First Patient Enrolled in Summit’s PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid in Boys With DMD Nasdaq:SMMT

First Patient Enrolled in Summit’s PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid in Boys With DMD Nasdaq:SMMT: "OXFORD, United Kingdom, June 17, 2016 (GLOBE NEWSWIRE) -- Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium difficile infection, today announces that it has enrolled the first patient in PhaseOut DMD, a Phase 2 proof of concept clinical trial of ezutromid (formerly SMT C1100) in patients with DMD. Ezutromid dosing is expected to follow a screening period of up to 28 days.

Ezutromid is an orally administered small molecule that is designed to modulate utrophin, a protein that is structurally and functionally similar to the dystrophin protein. Dystrophin is essential for the healthy function of all muscles but is missing in patients with DMD. Utrophin modulation is a potential disease-modifying approach that could treat all boys and young men with DMD, regardless of their underlying dystrophin gene mutation."



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Saturday, 4 June 2016

Enzyme protein neutrophil elastase may be key contributor to development of muscular dystrophy

Enzyme protein neutrophil elastase may be key contributor to development of muscular dystrophy: "Scientists at the University of Liverpool have discovered that muscle cells affected by muscular dystrophy contain high levels of an enzyme that impairs muscle repair. This finding provides a new target for potential drug treatments for the disease, which currently has no cure.

Muscular dystrophy (MD) is an inherited genetic condition that gradually causes a weakening of muscles. Duchenne muscular dystrophy (DMD) is the most common, and one of the most severe types, of the disease. There are around 2,500 people in the UK living with DMD, which usually affects boys in early childhood and leads to progressively worsening disability and premature death.

In DMD, the stem cells that normally repair damaged muscle are impaired, for reasons that remain unclear. In this new study, published in Scientific Reports, researchers looked at the molecular composition of the environment within which these muscle stem cells are found, to investigate whether this could be responsible for impaired function."



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Wednesday, 20 April 2016

Removing immunomodulatory protein improves symptoms of muscular dystrophy in mice

Removing immunomodulatory protein improves symptoms of muscular dystrophy in mice: "Removing an immunomodulatory protein called osteopontin improves the symptoms of mice with muscular dystrophy by changing the type of macrophages acting on damaged muscle tissue, according to a paper published in The Journal of Cell Biology. The study, "Osteopontin ablation ameliorates muscular dystrophy by shifting macrophages to a pro-regenerative phenotype" by Joana Capote and colleagues, adds support to the idea that osteopontin inhibitors could be used to treat patients with Duchenne muscular dystrophy (DMD).

DMD is a progressive, and ultimately fa"



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