One dose of the drug alters a child’s biology and improves muscle movement and function. (Image for illustrative purposes only).
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For the first few months of her life, Ayah Lundt was a healthy girl.
Born in January last year in Bornholm, Denmark, weighing 8.3 pounds, the girl grew normally and without problems during her first months of life.
At six months, he was crawling and trying new foods, with bananas and mushy broccoli being his favorites.
Nevertheless, everything changed after nine months. Upon reaching that age, she was unable to lift her head while lying down, sit alone, or clap, things I used to do normally.
A little Kenyan girl, Ayah Lundt, is hoping to receive the world’s most expensive drug which costs Ksh 230 million, for the treatment of a genetic disease, after Kenyans and the rest of the world joined hands in raising the money. https://t.co/xBdO52T4eO
— Kenyans.co.ke (@Kenyans) April 3, 2021
His legs began to weaken when he tried to crawl or stand. AND things got even worse when she couldn’t even eat oatmeal from a spoona, something he had done numerous times.
Ayah was diagnosed with spinal muscular atrophy, a genetic disease found in one in 10,000 children born worldwide.
He is now 14 months old and her parents are in a race against time to raise $ 2 million for a one-time treatment that could save your life before they are 2 years old.
The treatment is called Zolgensma, the problem is that it costs $ 2.1 million per dose, which is why it is known as the most expensive drug in the world.
“I think about the cost every day, and it weighs on me. And then I look at Ayah and see her get worse. As a mother, what would you do if you knew that there was something that could save your child? ”Said his mother, Mary Mithika, according to CNN.
Spinal muscular atrophy (SMA) is a progressive disease caused by a faulty gene.
The disease kills nerve cells and causes babies’ muscles to wear out, which can cause difficulty in swallowing or breathing.
As their muscles deteriorate, babies with SMA develop tremors in the fingers and the spine curves. They cannot stand or walk without assistance, and most do not survive beyond early childhood due to respiratory failure.
Zolgensma, the gene therapy treatment, is not a guaranteed cure. Ayah’s mother says the girl will never walk again. But a single-shot intravenous dose delivers a fully functional copy of the gene into nerve cells, altering the child’s biology and improving muscle movement and function, which would save the girl’s life.
Although the family is from Denmark, Ayah’s parents are raising money to treat her in the United States, where Zolgensma was approved in 2019 for use in children under the age of 2.
So far, the family has raised $ 1,200,000, thanks to a GoFundMe fundraising campaign, in which you can donate by clicking on the link.
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Eddie is an Australian news reporter with over 9 years in the industry and has published on Forbes and tech crunch.