I have asked my good friend Anna to do a guest post for me, to tell you the story of her two brave boys who suffer from a rare genetic disorder. We have been friends with Dave and Anna for over 20 years, and have enjoyed many fabulous summer parties at their home in Gloucestershire, and watched Archie and Isaac grow to be great boys, just another two kids in our group running riot at parties. But they need our help – over to Anna….
Aly has invited me to share our message here as we are fighting for treatment for our children who have a rare condition. The decision to provide the treatment on the NHS is getting bogged down in political bureaucracy and their drug is about to be taken away from them.
Archie (nearly 14) and Isaac (12) have Morquio Syndrome – they have physical disabilities which will get worse as they get older. When they were diagnosed there was no treatment. All we could do was keep our fingers crossed for medical advances but prepare for the fact that they would end up dependent on wheelchairs, with chronic pain and at risk of serious health problems. Exactly how hard and how quickly it would hit them would be down to chance.
But we didn’t let this stop us getting on with life, they are fab kids and have joined in everything at school, clubs and appearing on stage in panto!
The breakthrough came just over 5 years ago. The boys joined a trial of a new drug which might stop things getting worse. Hey, so the treatment had to be given at hospital in Manchester, only a 300 mile round trip from home. Oh yes, and it is given by an infusion which takes all day. And yes, every week. But it was a chance of better health for their future, so we did it. And it worked. The boys’ condition is stable, they are healthy and whilst still disabled, they do not have significant pain and can still walk.
So what’s the problem? Well, the trial finished in April last year, the drug was licensed and ready to be approved for use on the NHS. The drug company helpfully agreed to keep providing the drug whilst the NHS sorted the paperwork and so Archie and Isaac have continued the vital treatment, now given by homecare nurses during a day at school (yay!) But the NHS has not got its act together, the government reforms over the last couple of years have meant they do not have an effective process to make a short term decision to fund this kind of treatment for rare diseases. No new patients have been able to start treatment. And now the drug company have run out of patience and will not be funding the treatment after 12th May this year.
So we are fighting for a government decision before this date. All support welcome! Follow the campaign on facebook at https://www.facebook.com/archieandisaacsfightfortheirfuture and Twitter at @FundVimizim and find out how you can help by emailing your MP and signing our petition. #fight4treatment.
So please join us in supporting Anna’s cause and share the details of their campaign far and wide, and join me in wishing Archie and Isaac a long pain free and full life.