PRESERVE THE LIVES OF CHILDREN LIKE SAM AND KAMAL WITH MORQUIO DISEASE OVER THE NEXT 4 MONTHS WHILST NHS ENGLAND DECIDE THEIR FATE
Please help us to provide 4 months’ supply of Vimizim to all Morquio children and young adults, who have been let down by NHS England's failure to make a decision and fund Vimizim until 28th October 2015.
If 4 million people in England each gave £1: or 8 million people each gave 50p all the children and young adults with Morquio disease in England will be treated for 4 months until NHS England make their decision. Your donation will also send a huge message to the UK Government that being born with a rare disease should not mean discrimination against access to treatment.
34 UK children and young adults were human guinea pigs on the clinical trial development of Vimizim, a drug to prevent progressive disability and death in their twenties. After 5 years on the clinical trial and Vimizim being licensed for Europe on the 28th April 2014 NHS England have failed to make promised decisions and on 2nd July 2015 were told to wait another 4 months. These young people now face progression of their physical disease and early death.
The Prime Minister should be ashamed that he presides over an unaccountable quango called NHS England that has refused to make a decision on Vimizim when the Governments of 30 other countries including Turkey who are paying for Vimiizim for 176 children with Morquio disease. England led on the Vimizim clinical trial for Morquio disease bringing substantial financial benefit to seven NHS Hospital Trusts but when it comes to translating clinical research into clinical benefits English patients are cheaper dead than alive.
We recognise that this is not the usual natural disaster with all the visibility of the press behind it but it is an earthquake of ethical proportions for the people with Morquio disease in England.
Your donation however small will go towards funding Vimizim and supporting children and young adults with Morquio disease. It will also send a vital message to the Government that NHS are operating a discriminatory policy by denying children born with ultra-rare diseases treatment that can arrest their disease and enable them to live their lives to their full potential.
- For Sarah. treated, she will be able to continue studying for her PhD
- For Angela, treated, she will be able to continue to work without the worry on how she will pay her mortgage
- For Jibreel, having graduated with a BSc last year and will continue to progress in his career
- for Archie who has just selected his GCSEs he will need his treatment to pass his exams
- For Sam, he will continue to enjoy his childhood as every six year old boy should
And for the others like Sarah, Angela, Jibreel, Archie and Sam your generosity will play an amazing role over the next four months.
Your donation will also send the clear message to the Prime Minister, Secretary of State for Health and Members of Parliament that having an ultra-rare disease for which there is an effective but expensive licensed drug is not an excuse to condemn the small numbers of children with ultra-rare diseases to a life of progressive disabilities and death.
About Mucopolysaccharidosis IVA (Morquio)
Morquio is a rare inherited degenerative disease which affects 105 children and young adults in the UK. Patients with the condition appear healthy at birth, but within 24-36 months start to show symptoms of severe problems.
The condition is a form of mucopolysaccharidosis, a metabolic disorder caused by the absence or malfunctioning of lysomal enzymes. The signs and symptoms of Morquio can affect many different parts of the body and frequently include heart, lung, visual, hearing and dental complications in addition to the skeletal abnormalities and difficulty breathing.
Untreated individuals with Morquio deteriorate steadily resulting in diminished independence, decreased endurance, impaired quality of life and early death and can only be managed palliatively.
Vimizim is a life changing enzyme replacement therapy (ERT) drug that targets the enzyme deficiency in people with Morquio. Following an extensive clinical trial, it was licensed by the European Medicines Agency (EMA) in April 2014. Vimizim has improved individual’s stamina and energy levels and enabled children to continue growing taller than the average untreated individual with the same condition. Children have required less surgical procedures and are less reliant on wheelchairs and mobility aids allowing for greater independence.
Individuals with Morquio rarely grow to more than three feet tall. The licensed treatment Vimizim has been found through extensive clinical trials to slow down disease progression and associated symptoms resulting in a much enhanced life expectancy, improved health and quality of life.
Children who started on Vimizim at a young age are now only using wheelchairs occasionally to enable them to cover more distance or preserve their energy when outdoors, or at school or don't require them at all. Children's stamina and energy have increased enabling them to keep up with their peers and participate in clubs and activities such as swimming, football, drama, and playing independently which had the disease followed its normal progression would not have been possible. Children on treatment have grown taller than the average person with Morquio. Their physical condition has much improved and they rarely complain about pain. The younger children who started treatment have seen their bones growing more "normally" and many have so far avoided a number of the surgical procedures associated with the disease.
Young adults have also seen huge improvements in their health and stamina and are describing that pain is more manageable and that their body feels lighter and no longer like they are moving through treacle. Prior to treatment many were concerned about their deterioration and having to give up work or PhD study Vimizim has removed that fear.
Vimizim is the only treatment available for those with Morquio with no alternative apart from managing symptoms to make the individual as comfortable as possible and providing palliative care.