The Kok's sibling Ju Nyuk and Tze Bing might be the 2 earliest Pompe diagnosed in Malaysia, unfortunately they did not belong to the infant type which Myozyme (the drug to treat Pompe) was designed to. Therefore, they could not enroll into the trial offered by Genzyme (the company). But the brave mom Mdm Bong never gave up hope on them, she kept on looking for ways to get the medication for her kids. It was only around 2008/2009 that they were finally had a chance to start the treatment after countless appeal to various parties and finally approved by MOH.
Even though Joo Nyuk and Tze Bing were already wheel chair bound and on 24 hours ventilation, Myozyme still gave them new hope of life whereby their health condition did not deteoriate further. They now have more energy, they are more alert, for Tze Bing he has better appetite to eat. These might not be important to others, but to the patients, I would say it's a gift of life.
Why I shared this, I hope everyone of us will never give up on hope. Treatment right is everyone's right, be it the common diseases or the rare diseases like us. The difference between us and others is mainly the way the journey is designed. This is a very tough journey whereby you will be walking alone most of the time. Still we need to continue walking until we get what we want.
How can cost be the barrier to start ERT treatment?
We must buy this idea ourselves if we are walking this journey. We must make use of every opportunity to tell our friends and families that we need their support for a change. Only then, we stand a chance to improve the current medical system set up.