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Treatment hope for Otley boy with rare disease

Treatment hope for Otley boy with rare disease

Published at 7:16am 3rd September 2015. (Updated at 6:40pm 3rd September 2015)

The NHS could provide a key drug which the family of a 6-year-old Otley boy have been campaigning for.

Sam Brown has Morquio Syndrome, a rare genetic disease which affects just 88 people in England.

Sam's family have been waiting to find out whether the drug Vimizim will be approved by the NHS.

Now, the National Institute for Health and Care Excellence (NICE) has issued draft guidance, provisionally recommending that the drug should be used to treat the condition, if specific conditions are met.

Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE, said: "MPS IVA is a serious condition that severely affects both life expectancy and quality of life.

"We are therefore pleased to be able to provisionally recommend elosulfase alfa as a treatment option for people with this condition."

NICE has now launched a consultation into its latest report, which will run until midday on 23 September. 

The comments it receives will be considered at its next meeting in October, before it publishes final guidance to the NHS.

For the last 10 months, Otley's MP, Greg Mulholland, has been leading the cross-party, multi-organisation #FundOurDrugsNOW campaign which is demanding funding for the drugs Vimizim, Translarna for treating Duchenne Muscular Dystrophy and Everolimus for treating Tuberous Sclerosis Complex.

Mr Mulholland said: "I welcome the announcement that NICE are leaning towards recommending approval of the Vimizim drug. Sam Brown and the 87 others across England with Morquio Syndrome are now a step closer to securing supply of the drug.

"It is certainly not ideal that the decision date has been delayed and I call on NICE to confirm the new date urgently. So whilst the announcement from NICE is welcome, clearly this is not job done, so the campaign will continue until the new decision date is confirmed, the drug is approved and funding for it is released."

Christine Lavery, Chief Executive of the MPS Society, which supports those with Morquio Syndrome and their families, added: "We are relieved by the announcement from NICE HST today and are working hard to complete the requested Managed Access Agreement. However, we call on David Cameron to now show compassion to the children diagnosed too late to participate in the clinical trial who are deteriorating whilst they wait for the final positive decision.

"Individuals with Morquio and their families have been emotionally drained by the catalogue of errors that has led to the dragging out of the decision making process by NHS England and the changing of goals. The Morquio community have had 16 months of waiting to receive Vimizim and the fact that we are still months from the final decision is an appalling way to treat those in our society whose health is deteriorating before their eyes."