A Cookstown mum has made a heartfelt plea to Health Minister Jim Wells to do what he can to speed up the process to enable her daughter access to treatment which could dramatically change her life.
Seven year-old Aoife McGee, suffers from Morquio Syndrome, an extremely rare genetic disease with degenerative and life limiting effects.
Aoife, a pupil at Phoenix Integrated Primary School, has already undergone major spinal surgery in Manchester as well as permanent treatment to medicate early onset cardiomyopathy - a condition which causes her heart to work much harder than is normal for a child her age.
She also suffers from limited mobility due to skeletal problems and is unlikely to grow beyond 4ft tall.
Aoife’s mum Seanin, explained to the MAIL about a recent clinical trial which could help to slow the degenerative effects of Morquio Syndrome.
“There were clinical trials for a drug called Vimizim in England, ongoing for the last few years, those trials finished last year and were a resounding success and it was hoped that the NHS in England would fund this drug, however because of some things which happened legally, the NHS were forced to remove their drugs commissioning framework, which has directly affected us in Northern Ireland because we don’t have our own framework.
“Historically we have always looked at NHS England’s guidelines to make our own decisions. So as they don’t have a decision making process at the minute, it means we don’t either.
“From the perspective of patients in Northern Ireland, we are calling on Health Minister Jim Wells to see what other alternatives we may have. We believe that it is possible to link in with Scotland’s framework in order to utilise it and make decisions with regards to funding these treatments such as the treatment for Morquio Syndrome
“I am trying to drum up as much support as possible and appeal directly to Jim Wells and ask him what he plans to do, because at the minute we are sitting in limbo and something needs to be done, and it needs to be done now,” Seanin added.
When asked what she would think if she was given this medicine, Aoife told our reporter: “I think it would help me feel loads better.”
Seanin describes it as ‘simply unliveable’ to know there is something out there which can help her daughter but is being denied access to.
“When you get the diagnosis, like Aoife did in 2010, it is a very grave thing to absorb, extremely grave and then when you are given that hope that there is this treatment, it is a huge deal within the medical community.
“For years and years there has been no known treatment, nothing that can help Morquio, but now they have something and it is not just a minor treatment it is a pioneering treatment which can actually alter the course of the disease.
“It doesn’t cure it outright because that is not possible but it basically stops it at source. It means her condition won’t deteriorate any further really or very slowly.
Seanin added: “Our own Health Minister Jim Wells here has the power to actually look into alternatives for patients.”
If you would like to support Seanin and Aoife find them on Facebook and Twitter by searching for ‘My Morquio Life’.