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- Charlie jones
- BBC News
published photo, MEGANE WILLIS
John Hall, his wife Megan and their son Edward
The health service in England has agreed to pay the costs of treating a child with a rare disease, the world’s most expensive drug, after a humanitarian campaign by his parents.
Edward, who was only 10 months old, developed spinal muscle atrophy, which means he lacks a protein vital for muscle development and affects the nerves that emerge from the spinal cord into the spine.
Edward is expected to receive the new gene therapy for this disease, Zolgensma, which costs £ 1.79 million.
Megan Willis, mother of Edward, 29, commented on the success of the campaign, saying, “We feel very lucky, happy and at ease.”
Previously, Megan and her husband, John Hall, had expressed feelings of being left behind in their “race against time” to get their child the drug, which they hope will change their life.
Doctors treating him said he should be given a single drug as soon as possible containing an exact copy of the missing SMN1 gene, which causes muscle atrophy, to stop the progression of the disease.
The mother explained that the medication could make a difference in Edward’s condition and his ability to stand on his own two feet. She confirmed that she was ready to stage a protest if the decision was delayed for a longer period.
published photo, MEGANE WILLIS
Edward, 10 months old, suffers from spinal muscular atrophy, which means he lacks a protein vital for muscle development
Willis said: “We have been fighting for this drug for a long time. We can’t believe we have finally made it. We wish we didn’t have to wait that long.”
She added that “Edward will be finishing his first year in September, and the weather is the determining factor.”
Zolgensma is said to be the most expensive drug in the world, at a cost of £ 1.79million per patient, although the NHS has said it has negotiated an undisclosed discount.
When the health authority approved the use of Zolgensma last March, guidelines established by the National Institute for Health and Care Excellence stated that it should only be used for children under six months of age who had no already received no treatment, which denied Edward access to the drugs on him.
But the institute added a decision on whether to give the drug to other children on a case-by-case basis.
About 65 children are born each year with spinal atrophy in England. The disease causes muscle weakness and affects movement and breathing, which means most children do not live beyond two years without assistance.
Studies indicate that zolgensma has helped children breathe without a ventilator, sit alone, crawl and walk after just one treatment.
Edward, who was diagnosed at the age of two months, had been injected with another drug called Spinraza every four months at Addenbrooke’s Hospital in Cambridge.
Mother Willis said that she now looked forward to enjoying life with Edward for the first time without struggling to pay for the property that was “imposed” on them.
“Our goal is to make it work. I think for the first time we can really plan for its future,” she added.
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