Wednesday, 16 November 2016

Immune system plays important role in Duchenne muscular dystrophy, research reveals

Immune system plays important role in Duchenne muscular dystrophy, research reveals: "A new paper, co-written by faculty at Binghamton University, State University of New York, increases the understanding of Duchenne muscular dystrophy (DMD)—one of the most common lethal genetic disorders—and points to potential therapeutic approaches.

"The findings suggest that the immune system has an important role in the muscle disease of Duchenne muscular dystrophy," said Eric Hoffman, professor of pharmaceutical sciences and associate dean for research at Binghamton University's School of Pharmacy and Pharmaceutical Sciences."

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» GTx exploring product candidate as potential Duchenne treatment Action Duchenne

» GTx exploring product candidate as potential Duchenne treatment Action Duchenne: "GTx is exploring SARM, their lead product candidate, a selective androgen receptor modulator as potential treatment for Duchenne muscular dystrophy. 

Preclinical studies have confirmed the beneficial effects from SARMs in mice genetically altered to simulate Duchenne muscular dystrophy, compared to control groups. The Company continues to pursue a potential strategic collaboration with biopharma companies experienced in orphan drug development."

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Thursday, 6 October 2016

Sarepta Therapeutics Inc. (SRPT) -Summit Therapeutics PLC (ADR) (SMMT) Deal Is "Low Risk, High Reward": Wedbush

Sarepta Therapeutics Inc. (SRPT) -Summit Therapeutics PLC (ADR) (SMMT) Deal Is "Low Risk, High Reward": Wedbush: "Sarepta Therapeutics Inc. (NASDAQ:SRPT) announced on Tuesday, a partnership deal with London-based Summit Therapeutics PLC (ADR) (NASDAQ:SMMT), for the development of drugs to treat Duchenne muscular dystrophy. As part of the deal, Sarepta will use its expertise to help develop Summit’s utrophin modulator pipeline and its key experimental DMD drug ezutromid in exchange for its European rights and a Latin America option. "



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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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