Schoolgirl, 8, Is Only Child In UK Denied Therapy

A schoolgirl with a rare degenerative disease whose parents are battling for the right for her to be given an expensive drug therapy is the only eligible child in the UK being denied the treatment, it was claimed yesterday.

Hayleigh Reynolds, eight, suffers from Hurler Scheie syndrome, a disorder caused by a deficiency of an enzyme required to break down substances in the body.

The syndrome, also known as mucopolysaccharidosis (MPS), occurs in infancy and causes progressive physical, and in some cases mental, disability.

Her parents, Robert and Heather, from Barmulloch in Glasgow, were told by NHS Greater Glasgow and Clyde that it would not provide a gene therapy treatment for Hayleigh which costs more than £100,000 a year.

It said the Scottish Medicines Consortium had recommended against prescription of the drug Laronidase in NHS Scotland.

Mr Roberts, the janitor at St Cuthbert’s RC Primary School in Barmulloch, and his wife then raised a judicial review of the decision at the Court of Session.

Last week the health board’s lawyer told Lady Calton at the Court of Session that it would reconsider its decision not to prescribe Laronidase.

The review process got under way yesterday, but a final decision will not be reached until next month.

However, yesterday it was claimed Hayleigh is the only child in the UK eligible to be prescribed Laronidase but who is not receiving it.

Christine Lavery, chief executive of the MPS Society, said: “This treatment is approved by the Department of Health for treatment in England, and indeed patients in Northern Ireland get this treatment and in Wales they also get this treatment on a named patient basis, so actually Hayleigh is the only child in the whole of the UK with MPS1 Hurler Scheie disease who is eligible for this treatment and has been denied it.”

Hayleigh has been absent from school for a fortnight after an operation to drain fluid from her brain.

She told the BBC yesterday how the condition affects her life. She said: “Sometimes my arms and knees get sore.

“I can’t brush my hair at the back because my arm won’t reach.”

Mr Reynolds said he believes Laronidase will improve the quality of his daughter’s life.

He said: “It’s very annoying and frustrating because its taken more than three years to get to this stage.

“Apart from prolonging her life, it will make her feel a wee bit better too, not so tired.”
Her parents said as a last resort one of them may have to move to England with Hayleigh in order to qualify for the treatment.

Mrs Reynolds said: “We have contemplated myself and Hayleigh moving to England and splitting the family up. It’s so hard to think about.”

Mr Reynolds said: “Financially it would be a disaster, having to keep two houses going, so it is the last option.”

A spokeswoman for NHS Greater Glasgow and Clyde said: “We based our decision not to prescribe Laronidase on the detailed evaluation carried out by the Scottish Medicines Consortium, which recommended against its prescription in NHS Scotland.

“We are sympathetic to the plight of any family in this situation.

“We made a commitment before last Thursday’s court judgment to have this particular case further reviewed by three senior clinical staff based at the Royal Hospital for Sick Children, at which this young patient’s doctor will present his case.

“We remain committed to seeing this process completed by December 8.”