The mother of a six-year-old boy with a degenerative disease was “gobsmacked” after his school friends raised hundreds of pounds for the charity fighting to get him access to a life-changing drug.
A non-uniform day and cake sale at Surrey Square Primary raised £700 after parents stuffed ten pound notes in the collection tin last Friday in support of pupil, Harvey Brown.
Mum Vikki Brown, of Galleywall Road, has been “overwhelmed” with well-wishers and offers of support after the story of her brave boy appeared in the News last week as she continues to battle the NHS to get the drug her son desperately needs to live a longer, happier life.
Harvey has an ultra-rare degenerative disease called Morquio, which affects just 77 children in the UK.
The inherited illness is caused by an enzyme deficiency, which means Harvey’s skeleton will not develop properly. His organs will continue to grow but his weak bones won’t and he is likely to develop heart disease and breathing difficulties while gradually losing his vision and hearing. There is no cure for Morquio, and, untreated, patients like Harvey, rarely live beyond their twenties.
The first drug which could help Harvey live a longer, happier life is not being offered on the NHS yet, even though it was approved by the Eurpoean Medicines Agency in April last year.
Despite the trial for Vimizim being run in the UK, the life-changing drug is now being offered in ten different European countries while British children like Harvey sit and wait, deteriorating day by day.
Harvey was first diagnosed with the especially unusual form of Mucopolysaccharide disease (MPS) in December 2013, after the trial for Vimizim had already started. So while other children his age are already seeing the benefits from receiving the drug on the extended trial, he has to wait for the NHS to agree a process on assessing drugs for ultra-rare diseases.
“It’s heart-breaking sometimes when he wakes up in the morning when he can’t move,” Vikki told the News last week.
To support Harvey and his family in their fight for Vimizim, his friend Aaron Akande, organised a cake sale to be held on the blue-themed non-uniform day, which raised an impressive £150 towards the total.
“I always knew Harvey had something but I didn’t know it was that serious and that he wasn’t getting the help he needed. If I had the disease Harvey would help me,” said the ten-year-old.
Christine Lavery, Chief Executive at the Society for Mucopolysaccharide Diseases, which has been fighting for Morquio patients to have access to Vimizm for the last nine months, said “irreparable damage” is being done while children like Harvey wait for treatment.
“Children who were on the clinical trial and have been receiving Vimizim for a number of years have seen their condition stabilise, have increased mobility and are less reliant on wheelchairs and mobility aids which has enhanced their quality of life and lead to greater independence.
“Harvey did not choose to be born with Morquio, it is an ultra-rare genetic condition and without treatment life expectancy is around 25 years old. We are expecting an interim reimbursement decision from NHS England on the 25th June and will continue to do all we can to ensure that this is positive for Harvey and the other children and young adults desperate for treatment,” she said, adding thanks to Surrey Square Primary for their support.
When Vikki found out how much had been raised on the day she said: “I couldn’t believe it. I was so proud of the children and their families and gobsmacked at the amount raised.”
“I’d really like to thank everyone at Surrey Square Primary, Sue Lia, Karen Pallen, Ian Leppard and Katherine Pooch for their support,” she added.
A spokesperson for NHS England said they are currently assessing the results of a public consultation into their assessment process for ultra-rare disease drugs, which ended on April 23. “ It’d be wonderful to be able to fund everything and do so instantly, but unfortunately that’s never been the reality, so instead there is an annual process where the NHS carefully identifies the highest priorities for new treatments, which is what’s now happening,” said the spokesperson.
Until the end of June Vikki, 46, says she is just “hoping for the best” and doing everything she can to support the MPS Society, in their fight to get Vimizim for children like Harvey.
To make a donation or find out how you can add your support, visit www.mpssociety.org.uk
Read more about Harvey here