Newport Beach - July 31, 2012

In August, Coalition Duchenne will make the Malaysian state of Sabah the focus of its global efforts to raise awareness for Duchenne muscular dystrophy (Duchenne). The charity will hold its second annual Expedition Mt. Kinabalu on August 24-26, 2012, led by its founder Catherine Jayasuriya, whose grandmother was a Kadazandusun and whose 19-year-old son, Dusty Brandom, has Duchenne.

“The people of Sabah have been so supportive of our cause,” said Catherine. “Duchenne is the most common fatal childhood genetic disease. Now is the time for humanity to come together to help, and in Sabah we see that happening.”

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

Recently, Catherine appeared on Malaysian Radio and Television’s only English Talk Show “Hello on Two” to share the message of Duchenne awareness. Hosts Azaria Tagaya and Zaleha Khairene responded to Catherine’s call for global awareness and so did viewers.

“This year we have 60 climbers from around the world participating in Expedition Mt. Kinabalu,” said Catherine. “There is a strong Malaysian contingent. Many have never heard of Duchenne and now they are supporting the cause by challenging themselves to reach the summit of 13,455ft!”

Dusty Brandom, Catherine’s 19-year-old son who has Duchenne, knows about “physically” contributing to the cause. Dusty is part of a United States Food and Drug Administration (FDA) Phase 2b trial of a new drug called ataluren developed by PTC Therapeutics. Dusty has travelled all over the United States, endured four muscle biopsies and withstood hundreds of blood draws in support of the trial. His toil will largely benefit the next generation of boys with Duchenne. However, he is excited that the drug is moving forward towards FDA approval. PTC Therapeutics recently announced the initiation of an additional open-label study in the European Union, Israel, Australia and Canada.

"The new study is based on the potential ataluren has shown in previous studies," stated Stuart W. Peltz, PhD, CEO of PTC Therapeutics. "We are encouraged by the results we have obtained in our Phase 2b study and are committed to the continued advancement of the development of ataluren," said Peltz.

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 sponsored research agreement with Phrixus Pharmaceuticals, a Michigan based biotechnical company to advance Carmeseal, a poloxamer cardiac and pulmonary treatment for Duchenne and heart failure. The Coalition Duchenne grant was a precursor to funding of over $600,000 by the US National Institutes of Health.

“Twenty thousand boys are born each year with Duchenne, more than 50 each day. Most only live into their 20s. We lose many in their teens.” said Catherine. “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.”

Dusty Brandom’s younger brother, 17-year-old Lucas, hopes that his efforts will help change the disease. Lucas was recently featured in newspapers in California as he and two friends, Evan Romano and Ian Henderson, prepare to travel to Malaysia for Expedition Mt. Kinabalu.

“This is my second time climbing Mt Kinabalu to raise awareness for Duchenne,” said Lucas. “When I see my brother, who cannot even comb his own hair, I am grateful to have my health, and it’s important to help those who do not. Dusty and I used to be in a band together but now he isn’t strong enough to hold a guitar.”

Other Malaysian and regional newspapers have been supportive of Coalition Duchenne and have run feature articles. Catherine has been lauded as a Malaysian who is making a difference on the world stage. This month, travellers on Dragonair will read of her efforts in their inflight magazine Silk Road. Catherine will also be featured in KL Lifestyle magazine.

“Malaysians can make a difference when they think globally and act locally,” said Catherine. “There are many boys and young men in Malaysia who will benefit from our efforts to raise awareness and funding for research. Families here in Malaysia are devastated by a Duchenne diagnosis, as they are in Australia, the United States, and all over the world. U.S. Olympic swimming champion Ryan Lochte, who is in the news this week for winning gold, lost a family member to Duchenne, and is an advocate for raising awareness for Duchenne.”

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 US 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,” said Catherine.

Catherine, Dusty and their family met with US President Barack Obama in 2011 and shared with him facts about the disease. They also gave him a Kadazandusun sompoton as a gift and he tried to play the instrument.

“At first he played the sompoton upside down,” said Dusty. “It is hard to tell the President he is doing something wrong.”

Last month President Obama signed a new law called the FDA Safety and Innovation Act which will fast track development of new drugs to treat rare diseases like Duchenne.

“We are very thankful for the President’s focus on helping people like me with rare diseases,” said Dusty.

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 be part of the international movement raising awareness and funding for Duchenne contact Catherine Jayasuriya at Catherine@coalitionduchenne.org.


For more information about Coalition Duchenne, please visit www.coalitionduchenne.org. Donations can be made on the website at or at http://www.active.com/donate/duchenneexpedition2.

For the newspaper article featuring Lucas Brandom and Evan Romano see http://articles.dailypilot.com/2012-06-09/opinion/tn-dpt-0610-apodaca-20120609_1_duchenne-gabriella-dusty-brandom.

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

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

For more information about PTC Therapeutics, please visit www.ptcbio.com.