(From left) Dr Thong Meow-Keong, founding president of MLDA, Mr Lee Yee Sang, Minister of Health Datuk Seri Dr Dzulkelfy Ahmad, and Mr Lau Beng Long, Managing Director, Sunway Group Healthcare Services.
While the Ministry of Health has increased the number of activities to help create awareness about rare diseases as well as funding for the treatment of rare disease, without the cooperation of the private healthcare sector, researchers, and the public, we will not be able to achieve meaningful impact for those who are suffering from these rare diseases,
– Minister of Health, Datuk Seri Dr Dzulkefly Ahmad
Sunway City, Mar 3 – Thousands of runners, some in wheelchairs and with faces covered in fluorescent paint to indicate their support for patients with rare diseases, convened to run a unique five-kilometer route throughout Sunway City, to raise awareness for rare disease patients.
Organised by the Malaysia Lysosomal Diseases Association (MLDA) in partnership with Sunway Group and EC Pixels, the colourful charity run was officiated by the Minister of Health, Datuk Seri Dr Dzulkefly Ahmad, who also ran with hundreds of rare disease patients and their families in the first kick-off of the run. Joining him were Subang Jaya assemblyman Michelle Ng, Kinrara assemblyman Ng Sze Han, management of Sunway Group and the management of EC Pixels.
The run was held in conjunction with World Rare Disease Day, which takes place on 28 February every year around the world. This year’s theme is ‘Bridging Health and Social Care’, focusing on bridging the gap between medical, social and support services to address the challenges faced by rare disease patients and families.
Mooted by MLDA, ‘Run for Rare’, which took place for the first time in Malaysia, intends to create awareness for rare diseases. Additionally, runners were encouraged to participate in games stations which gave runners a chance to step into the shoes of a rare disease patient, with activities that simulated difficulties faced by patients in their daily lives.
The four game stations featured challenges such as a wheelchair obstacle, using a dumbbell weighted brush for calligraphy, enduring a tickling session with feathers and drinking special formula milk.
Dr Dzulkefly commended the successful partnership between NGOs and the private sector which had brought the runners and stakeholders together to raise awareness.
“While the Ministry of Health has increased the number of activities to help create awareness about rare diseases as well as funding for the treatment of rare disease, without the cooperation of the private healthcare sector, researchers, and the public, we will not be able to achieve meaningful impact for those who are suffering from these rare diseases,” he said.
MLDA founding president IR Lee Yee Seng, who has two children afflicted with Pompe disease, a type of lysosomal storage disease (LSD) which results in a rare neuromuscular disorder, had formed MLDA in 2011 with a group of parents who believed that they were the best advocates for their children who are suffering from LSD. MLDA currently has 90 LSD patients.
“With this first Run for Rare, we have managed to raise RM 100,000 and we will be using these funds to benefit LSD patients who need medical support and care. Some of these funds will be used to acquire an enzyme to treat these children on short term basis before pharmaceutical companies take over under a humanitarian programme, while waiting for government reimbursement. This is a life-saving journey, as without treatment, the patients will die.”
Lee added that patients live with various complications from muscle weakness to organ failure. Due to the rarity of their diseases, patients often receive erroneous diagnosis or wait years for an accurate diagnosis. The lack of clinical expertise and availability of treatment options often have devastating results on patients and their families. To make things worse, the cost of treatment such as ERT is very steep, up to RM1 million a year.
Despite these challenges, Lee said that he has seen progress. “In 2011, only 20 LSD patients were able to get medication. However, now with various humanitarian programmes and through collaborating with the Ministry of Health and industry partners such as BioMarin and Sanofi Genzyme, we are proud to declare that more than 50 LSD patients are currently being treated.”
Lee hopes to make the “Run for Rare” an annual affair to raise awareness and bring stakeholders together to alleviate the suffering of rare disease patients and their families.