Coalition Duchenne Announces 2nd Annual Expedition Mt Kinabalu

International Team of 60 Climb 13,455 Foot Peak, to Raise Funding and Awareness for Duchenne Muscular Dystrophy

Newport Beach - May 24, 2012

Coalition Duchenne will hold its second annual Expedition Mt. Kinabalu, a global event to raise awareness for Duchenne muscular dystrophy, on August 25, 2012. Building on the success of its 2011 event, an international team of 60 will climb Borneo’s Mt. Kinabalu, in the Malaysian state of Sabah. The climb will be led by the Coalition Duchenne founder Catherine Jayasuriya, whose grandmother was a Kadazandusun and whose 19-year-old son Dusty Brandom has Duchenne. At the summit of 13,455 feet, the team will raise the Coalition Duchenne flag in honor of the hundreds of thousands of boys and young men worldwide who have Duchenne.

In addition to raising global awareness, Coalition Duchenne’s fundraising events such as Expedition Mt. Kinabalu and Music Power (held in California in November 2011), have raised money that is funding much needed research to treat the disease.

Coalition Duchenne’s research focus is on cardiac and pulmonary initiatives. Cardiomyopathy and the loss of the ability to breathe independently often cut the lives of those with Duchenne tragically short.

In March 2012, Coalition Duchenne announced the funding of a $25,000 sponsored research agreement with Phrixus Pharmaceuticals, a Michigan based biotechnical company to advance Carmeseal™ a poloxamer cardiac treatment for Duchenne and heart failure and a potential skeletal muscle enhancer.

Professor Joseph Metzger Ph.D., chair of Integrative Biology and Physiology at the University of Minnesota, conducted experiments that showed the treatment could boost the blood pumping capacity of damaged hearts by binding to the damaged regions of cell membrane.

When Carmeseal is infused into the bloodstream it encounters and binds to microscopic tears in the heart muscle and possibly skeletal muscle. This may prevent the pathological leak of calcium into the heart cells, which could cause calcium overload and keep the heart from delivering sufficient oxygenation to the vital organs. Carmeseal, which has been shown to be effective in four animal models of DMD and heart failure, is expected to have its effect in patients with DMD irrespective of the genetic defect that causes the disease. The treatment is potentially applicable to the larger population of patients with cardiomyopathy.

“20,000 boys are born each year with Duchenne, more than 50 each day. Most only live into their 20’s. We lose many in their teens,” said Catherine Jayasuriya. “We need to focus on changing the course of the disease. Developing Carmeseal is one way we will eventually change that outcome.”

Coalition Duchenne has also joined other non-profits in funding the anti-fibrotic drug HT-100 being developed by Halo Therapeutics, a Massachusetts based biotech. HT-100 works by inhibiting the pathological fibrotic process in muscle and directly stimulating healthy muscle fiber regeneration.

As Catherine states, “Duchenne is a difficult road, and presents significant challenges along the way. Duchenne steals many things, the ability to walk, to hug, to move, to talk, to breathe, but there is that something inside of you, that it can never get to, that it can never take...Hope.”

Coalition Duchenne is also hoping to advance the application of stem cells to cure ailing hearts. Coalition Duchenne is currently working with Cardiac researchers at Cedars Sinai Hospital in Los Angeles California to investigate the applicability of cardiac stem cells to Duchenne. Coalition Duchenne is seeking to sponsor researchers to build on the work of Roberto Bolli at the University of Louisville in Kentucky recently published in Lancet. Dr. Bolli improved heart function and reversed fibrosis using cardiac stem cells in patients with heart failure.

Catherine founded Coalition Duchenne in 2011 to raise global awareness for Duchenne muscular dystrophy, to fund research and to find a cure for Duchenne. Coalition Duchenne is a 501c3 non-profit corporation. Coalition Duchenne is a call to action; it is about bringing not just Duchenne charities together, but everyone. It calls for humanity to come together as nations, as people, as individuals, to help. As Martin Luther King Jr. said, “We are confronted with the fierce urgency of now.”

Duchenne muscular dystrophy is a progressive muscle wasting disease. It is the most common fatal disease that affects children. Duchenne occurs in 1 in 3,500 male births, across all races, cultures and countries. Duchenne is caused by a defect in the gene that codes for the protein dystrophin. This is a vital protein that helps connect the muscle fiber to the cell membranes. Without dystrophin, the muscle cells become unstable, are weakened, and lose their functionality. Life expectancy ranges from the mid teenage years to the mid 20's. Their minds are unaffected.

To join the climb and to be part of this international movement to raise awareness for Duchenne, please contact Catherine Jayasuriya at Catherine@coalitionduchenne.org.

Donations can be made on the website at or at http://www.active.com/donate/duchenneexpedition2.

For more information about Coalition Duchenne, please visit www.coalitionduchenne.org.

For more information about Phrixus Pharmaceuticals, please visit www.phrixuspharmaceuticals.com.

For more information about Halo Therapeutics, please visit www.halotherapeutics.com.

For a summary of the stem cell research by Dr. Bolli, please refer to "Repairing Broken Hearts." The Economist 19 November 2011: 88-89.