This little sunshine is Leong Xian Yi and was born on 18th June 2014. This year she will be 4 years old and she loves to go to school. She is a cheerful, bubbly, happy girl who love to dress up like a pretty princess every day. Generally, everyone at home addresses her as Princess Mei Mei. Little did we know, behind every happy smile, she is slowly developing a symptom of life-threatening rare disease.
On 16th January 2018, she was diagnosed with a rare disease known as Morquio Syndrome A (MPS IVA ). An estimated 1 in 200,000 to 300,000 live births have a diagnosis of Morquio Syndrome A.It is a rare metabolic disorder whereby the body could not process the long chains of sugar molecules that are found through the body (often in mucus and in fluid around the joints). This missing enzyme function is to breaks down or recycles materials the body can’t use. When the body does not produce enough of this enzyme, toxic will build up in the tissues, bones, and major organs. The build-up of toxics called glycosaminoglycan (GAGs) and it will cause serious problems, including heart disease, skeletal abnormalities, vision and hearing loss, difficulty breathing, and early death.
Mei Mei appear to be healthy at birth, but at the age of 1.5 years old, we slowly observe the abnormality on her skeletal system; short stature, knock knees and abnormalities of the ribs, chest, spine, hips and wrist. According to her doctor, Mei Mei’s eye (cornea) will typically become cloudy as she grows which can cause vision loss and recurrent ear infection which might cause hearing loss. Life span of Morquio patients depends on the severity symptoms and they may survive until late childhood of adolescence.
Currently there is NO CURE for this disease, and the only treatment that can be done is to slow down Mei Mei’s condition from further deterioration is Enzyme Replacement Therapy (ERT). This enzyme is a type of drug called “Vimizim” (elosulfase alfa), which was approved by FDA in 2014 and subsequently made available in Malaysia in 2015. Weekly infusion of Vimizim is required to help break down some of the long chains of sugar molecules called glycosaminoglycan (GAGs) in Mei Mei’s body. It works on a cellular level to restore cell function. It is not a cure, nor does it help with her bones, but we were told it can buy Mei Mei some time to live like a normal child again until some other cure is available. The only blessing is that besides the physical shortcomings, patient impacted by Morquio like Mei Mei is expected to have normal intellectual capability.
Based on our consultation with the doctor, the estimated total cost of ERT for Mei Mei is approximately RM 1 million (USD 250k) per year and continuous treatment is needed for the rest of her life. We were obviously shattered when we heard about the cost involved. We felt helpless as the estimated total medical expenses for Mei Mei is way beyond our affordability. Most insurance coverage would not typically cover these diseases. We believe this cost would be overwhelming for middle class families in Malaysia. I am working as a barber, while my wife is a homemaker, supporting a family of 5.
We are starting this fund-raising campaign hoping for your kind blessing and charitable donation to support Mei Mei’s medical treatment and specialist consultation. We greatly appreciate everyone’s help and moral support throughout this difficult period for our Princess Mei Mei. Nevertheless, we realize is an incredible thing to ask, but if you could help in anyway even by just sharing this post to bring social awareness and acceptance of the rare diseases itself will be much appreciated.
There are no words to describe our gratitude towards your kindness other than a sincere Thank You & May God Bless you from the bottom of our hearts. What we are trying to achieve for Mei Mei is to alleviate the impact of Morquio, while praying for the long-term cure to arrive. We are uncertain about her future, we know that it would be extremely challenging, but as parents, we ought to try our best to make the best of the road that lies ahead of her.
On behalf of our family and Mei Mei, thank you.
(Leong hon thong & Low siow ching )
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PUBLIC BANK >>6447059804<< LEONG HON THONG/LEONG XIAN YI
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