Many MPS-II patients now have some hope

17/12/2013 23:27

I am grateful that out of all the companies that develop drugs for MPS sufferers, Shire is the only one that has developed a treatment (Elaprase) for Hunter Syndrome. Though not a cure, it does extend the life of patients, and gives most a better quality of life.

I know Shire is a business and all businesses need to make a profit to survive but they are also helping the many patients who would have otherwise have no hope. I thank them for their dedication to finding a treatment and investing their own hard earn funds in researching a cure for this degenerative condition. I applaud their efforts and encourage them to continue forging ahead. I do remind myself I am not entitled to treatment just because my son has a condition. Frankly Governments around the world do not have the available budgets to fund medical research and clinical trials, hence I am very thankful that we have companies such as Shire that are willing to fund research into rare diseases. I encourage them to be prudent in their decision and be speedy as many life depends for the most part on their good will.

I do know that sometimes more is achieved through patience, diplomacy and respect. As a mum and person that advocate for fairness we were able to outline a case for the many benefits to Shire financially and brand wise for them to consider Australia for the phase III trial. Of course there are still many more hoops to jump through but things are certianly positive and moving in the right direction.  However, we also know there is a protocol for which patients are recruited and a strict criteria in qualifing to take part in the study. This process of course can be grueling, exhausting and may even seen unfair because of the strict criteria. However, I do believe the trial is worth the effort. Therefore all stakeholders need to work together for the safety and viability of the long term future of the study.   

And let all be given a ‘fair go’ to benefit from the glimmer of hope!