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Megan Willis felt a “punch to the stomach” when she learned that the only 8-week-old baby she has with her partner John Hall suffers from a rare musculoskeletal disorder.
“It’s unthinkable, it’s very painful to handle,” says a 29-year-old mother from Essex, England, about what little Edward, who suffers from spinal muscle atrophy, a rare disorder caused by a lack of a protein that is vital for muscle growth and movement.
Until 3 years ago, there was no cure for the most serious form of the disease, which the baby suffers from. The condition is even fatal, as the muscles atrophy causing respiratory problems and death, usually before the age of 2.
But now there is hope, although it is tremendously expensive. THE injection that can stop the rapid deterioration of the baby costs 7 1.7 million.
The baby who could be saved with a 1.7 million pound injection https://t.co/Eji4k8ZRk7
– Daily Mail UK (@DailyMailUK) December 15, 2020
Zolgensma is believed to be the most expensive drug in the world, as it replaces a faulty gene.
In a 2017 study published in the New England Journal of Medicine, 15 babies who received Zolgensma were alive at 20 months of age. That is where only 8% of babies reach this age.
In England it is not currently administered by the national health system, as is already the case in Germany, the United States, Japan and Brazil.
The only way to give Edward a chance is for his parents to find the money and travel abroad. The global funding campaign has raised more than .000 120,000, a large amount but a small percentage of the money needed.
Zolgensma is only the third gene therapy approved in Europe and of course not the only one so expensive. The first, which received the green light in 2012, was the .000 750,000 Glybera and the second the Luxturna, which cost 13 613,000.
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