Arthur Morgan, 5 months old, suffers from a disease that kills his muscles. Each year, only 60 children worldwide suffer from this deadly disease. More than half of children with this disease called Spinal Muscular Atrophy do not survive more than two years, but a few years ago there was hope to gain it and today its treatment is possible with the most expensive drug. of the world. . Many improvements have been seen in children with a single dose and it is expected that in the times to come, its benefits will be even more …
This drug can save lives
Arthur, Reece and Rosie’s son, also had difficulty sitting up and keeping his head straight. Three weeks later, she was given Zolgensma gene therapy. Made in America, this drug is considered the most expensive drug in the world. The cost of a single dose is over £ 17 lakh, or around Rs 16.9 crore. Studies have shown that it can protect against paralysis. It is given by intravenous infusion and produces a protein that is not produced in people with SMA.
What is this disease?
The British National Health System (NHS) has partnered with Novartis Gene Therapies for this. AMS is a disease in which type 1 muscle is lost. In this, the body stops producing a protein called SMN which is necessary for the growth and movement of muscles. Over time, the muscles in the chest begin to wear down, making it more difficult to breathe and making it difficult for the child to live beyond the age of two.
Medicine given to Arthur
I received treatment 3 years ago
About three years ago, in a 2017 study published in the New England Journal of Medicine, it was found that the 15 children who received this injection could survive for 20 months, compared to only 8% of children in previous research. . treaty. 12 out of 15 children received higher doses and at 20 months 11 could sit up unaided and two could even walk. The most important thing is that it does not require putting them on a ventilator. This treatment was invented recently, so you will have to see its results later.