The parents of a dying baby have succeeded in their son’s campaign to be treated with the most expensive medicine in the world.
The new gene therapy, Zolgensma, will be used to treat 10-month-old Edward from Colchester for severe spinal muscle atrophy (SMA) after his parents received a green light earlier this week. A genetic condition caused by a lack of protein weakens the muscles, affects movement and respiration.
About 65 children are born with SMA in England each year, most of whom do not live beyond the age of two without intervention.
Edward’s mother, Megan Willis, who campaigned for the treatment of her, 1.79 million baby’s patient, told the Guardian she was “thankful and excited” to receive the news on Wednesday.
“I am exhausted. It has come a long way, “said the 29-year-old. “When I was diagnosed again, returning to myself in November, I did not think there was an alternative for him. I thought he was dying because he said all this when I read the SMA that 95% of children die or are severely disabled. “I did not think he had a future.”
The drug contains a copy of the missing SMN1 gene, allowing it to stop the progression of the condition. Willis said the family had a “glimmer of hope” after learning of the treatment, but felt overwhelmed when they learned of the cost of the treatment. After a “countless sleepless” nights, he was greatly relieved to learn that the NHS would refer to Edward in mid-August.
“Because the drug is so much more effective when taken at a young age, ‘time is of the essence,'” Willis said. “Ol olgensman just transforms the baby. Children walk, talk, eat, stand. That’s unbelievable. “
Edward, who was diagnosed within two months, is currently being treated for Spinraza, which is injected every four months. Unlike a single injection of Zolgensma, it is a lifelong treatment.
Zolgensma was licensed for use by the NHS in March, but guidelines set by the National Institutes of Health and Excellence stated that it should only be used for children under six months who are no longer receiving treatment. However, it added that a decision on whether to give it to other infants, including those who love Edward’s other treatments, will be considered on a case-by-case basis.
“We are so lucky,” Willis said. “Edward has a future, before that many of his children had no alternative. We are in a new era of SMA. The fact that it is an option for our children is a blessing. ”
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