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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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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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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