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Annual Expedition Mt. Kinabalu Raises Awareness of a Duchenne Clinical Trial Bringing Treatments to Boys in Malaysia
Date:7/25/2018

A pioneering Duchenne muscular dystrophy clinical trial is now recruiting patients at two sites in Malaysia, and Coalition Duchenne dedicated its annual Mt. Kinabalu climb to raising awareness of the need to enroll patients.

“There is no cure for Duchenne, and the journey to finding one starts with patients and families willing to participate in a clinical trial,” said Catherine Jayasuriya, the founder and executive director of Coalition Duchenne, a global charity committed to raising awareness and funding treatments for the disease. “This year, our expedition is dedicated to raising awareness of the new opportunities for families to enroll in clinical trials in Malaysia.”

For the past eight years, Jayasuriya has led climbers up Malaysia’s highest peak to raise money and awareness for Duchenne and the devastating impact it has on patients and families. Collectively, the annual expedition has brought together nearly 500 people from all over the world to climb Mt. Kinabalu. More than two dozen climbers made the trek for this year’s annual climb, from Germany, the UK, Australia, Malaysia, the US and elsewhere, held July 21-23, on the island of Borneo, in the Malaysian state of Sabah.

“Our climbers trek up the mountain for all the brave and courageous boys and young men who can’t, and to honor those who have lost their lives to Duchenne,” Jayasuriya said.

Jayasuriya and her family speak about the importance of clinical trials from personal experience. Their son, Dusty Brandom, now 25, was one of the first patients to enroll in a PTC Therapeutics’ clinical trial for ataluren, an orally administered medicine for the treatment of Duchenne caused by a nonsense mutation.

PTC Therapeutics, a US-based biopharmaceutical company, is the sponsor of the clinical trial currently recruiting Duchenne patients at two sites in Malaysia and other countries. Investigators are looking for boys with Duchenne aged five and above who still have the ability to walk. The study will examine whether ataluren can delay Duchenne progression and maintain patients’ ability to walk, run, and climb up and down stairs over a period of 144 weeks (2.8 years). The safety of ataluren will also be assessed.

The Principal Investigators at the two clinical trial sites in Malaysia are Teik Beng Khoo, MD at Kuala Lumpur Hospital, and Meow Keong Thong, MD at University Malaya Medical Centre.

It is hoped that the results of this trial will drive better understanding of Duchenne and its treatment, and, ultimately, benefit future patients.

Joining this year’s Expedition were two representatives from PTC Therapeutics from the United States.

Jayasuriya grew up in Kota Kinabalu and always dreamed of hiking up Mt. Kinabalu with her children.

“I’ve been to the summit with so many people but never with my son Dusty,” Jayasuriya said. “It is heartbreaking that his body has failed him, but his mind is full of hope and dreams. Dusty is my daily inspiration and he inspires so many people with his ability to overcome personal adversity. This year, as I do every year, I climbed for him.”

In addition to the annual climb, Jayasuriya has talked about her advocacy work to physicians around the world, most recently at the 2017 World Orphan Drug Congress in Singapore. She advocates for Asian patients to have access to drugs being developed in the US and Europe.

“Boys and young men with Duchenne in this region need a voice and access to new drugs,” Jayasuriya said. “The drug trial participants are so brave and contribute significantly to the advancement of treatments for Duchenne and other diseases.”

Dusty and his grandfather, Tan Sri Thomas, are featured in Jayasuriya’s award-winning film Dusty’s Trail: Summit of Borneo (2013). The film is the story of Dusty’s journey with Duchenne, and it tells about people coming together from around the world to climb Mt. Kinabalu to raise awareness. Dusty’s Trail has an important message of hope in the face of adversity, and shows the beauty of Sabah and its people to the world.

About Coalition Duchenne

Catherine Jayasuriya started Coalition Duchenne with her son Dusty Brandom 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 and its founders have been leaders in sponsoring Duchenne research for over 16 years. Coalition Duchenne has several groundbreaking research initiatives that are making advances in potential cardiac and pulmonary treatments for sufferers of Duchenne and other muscle weakening conditions. The charity is one of the leading advocates for FDA approval of new treatments for Duchenne.

Coalition Duchenne recently launched Duchenne Without Borders, which supports underserved families and boys with Duchenne. Duchenne Without Borders provided boys with Duchenne in Sabah with Ambu bags and bipap machines, and provided information about Duchenne to doctors and families.

The vision of Duchenne Without Borders is to expand its outreach to underserved communities around the world, and give hope and a connection to treatment protocols, drugs, and research initiatives that are readily available in the United States and other developed countries. Duchenne Without Borders has already established collaborations in India, Algeria, Cambodia, and the Philippines.

For more information about Coalition Duchenne: Please visit http://www.coalitionduchenne.org.

About Dusty’s Trail: Summit of Borneo (2013)

A press kit is available for download from the Dusty’s Trail website:
dustystrail.com

About Duchenne Muscular Dystrophy

Primarily affecting males, Duchenne muscular dystrophy (DMD) is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk as early as age ten, followed by loss of upper body strength. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and twenties. More information on the signs and symptoms of DMD can be found at: http://www.duchenneandyou.com

Read the full story at https://www.prweb.com/releases/annual_expedition_mt_kinabalu_raises_awareness_of_a_duchenne_clinical_trial_bringing_treatments_to_boys_in_malaysia/prweb15650024.htm.


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