THE PROBLEM “Without enough money to buy medications, doctors told me I have to choose which one of my two kids will get the medications and which one will live longer. How can a parent make a decision like that?!” . My name is Adam an I am 10 years old. I have a 6-year old brother named Arman. Both of us have been diagnosed with a rare disease called Mucopolysaccharidosis type II (MPS II) or commonly known as Hunter Syndrome. It is a rare genetic disorder that occurs when an enzyme your body needs is either missing or malfunctioning. This enzyme’s job is to break down certain complex molecules and without enough of this enzyme, the complex sugars called Glycosaminoglycans (GAGs) accumulate in the body in harmful amounts. The MPS build-up massive amount of harmful substances causes permanent, progressive damage affecting appearance, mental development, organ function, and physical abilities. We are unfortunate as MPS or Hunter Syndrome is only diagnosed in every 150,000 births. I was diagnosed with Hunter Syndrome at the age of 5 while my brother was diagnosed when he was 1 year old. It all started when I had constant flu at the age of 4 that I had to undergo tonsil surgery. Even so, I didn’t recover from the flu. Then, my mother started to notice some delays in my development, including my speech and decided to bring me to speech therapy, but I never able to talk. My parents were getting truly concerned while at the same time my health was uncertain. So they brought me to the genetic expert in University of Malaya Medical Centre, Prof. Dr. Thong Meow Keong and they were told by the doctor about the rare syndrome. The child may feature short stature and slowly experience loss of hearing, loss of sight and respiratory problem. Physically, the child will develop claw hands and bended legs due to joint stiffness. Without proper treatment, the child may have a heart failure that causes damage or even early death.
THE SOLUTION The Treatments and Costs The doctor advised my parents for us to undergo the Bone Marrow Transplant (BMT) which is quite risky with 70% fatality or to go for enzyme replacement treatment (ERT) that would give me and Arman a chance to live a normal life. However, ERT is expensive and beyond my parents’ ability to bear with. Each one of us requires RM6,000 per week for ERT treatment, equivalent to RM 24,000 per month. That means, my parents need to spend more than RM 500,00 every year in order to keep both of us alive or we may not survive through adulthood. We would like to have at least a secure funding of RM 288,000 for 6 months so that we can continue to go to school and play with our friends instead of being in a hospital bed.
THE IMPACT Rubee and her husband, parents of Adam and Arman, live in a constant concern and at times went through a depression when they found out that both of their children were diagnosed with MPS or Hunter Syndrome. They tireless continue their struggle to meet the weekly treatment costs to ensure both kids lead a life of a normal child. Sometimes, due to financial constraint, they had to make a decision to skip treatment for one of the kids and to give way to another. Missing a treatment is not an option and it broke their hearts when they had to choose one kid over another for treatment while they know the risk.