Little Edward, born in the county of Essex, in the south of the United Kingdom, was diagnosed at two months of age with spinal muscular atrophy (SMA). For his treatment, he needed a drug that costs around 2.1 million dollars and he obtained it through donations, thanks to the support of many people.
Several months have passed, with barely 1 year old, the little boy’s life took an impressive turn, comparable, according to his mother, to having “a new life”.
And it is that, due to the spinal muscular atrophy that he suffers, this British baby lacks a vital protein for muscle development.
This disease causes muscle weakness and affects movement and breathing, so most children who suffer from it do not live more than two years if they do not receive adequate treatment slogan the BBC.
In Edward’s case, he received it last August when, through a campaign carried out by his parents, he had access to a new gene therapy with the drug Zolgensma, known to be the most expensive drug in the world, at a cost of 2 , 1 million dollars.
With this treatment, Megan Willis, the mother, said her son has achieved milestones that she never thought would be possible.
For example, he pointed out that the minor, now, can turn around, sit alone. In addition, it has taken its first assisted steps.
“We are very proud of Edward. He’s doing incredibly well. It is far exceeding our expectations. We are incredibly lucky. All I ever wanted was for him to be able to sit down and then I knew he would have an amazing life, ”Megan said.
“We have been working on her gait for a few weeks and she moves her own legs in the leggings, which is a really good achievement. Tears welled up in my eyes when I first saw him. I thought ‘this is how it should be’, it was an incredible moment, “he added.
The family moved to London so Edward can receive physical therapy up to five times a week. Willis, who documents the progress on Instagram, stated that the little boy’s energy levels have improved and he went from being lethargic to being a normal, cheeky and playful baby.
“This medicine has brought Edward back to life. He has a new life. It is a marathon, not a sprint. It’s going to take a long time for Edward to catch up, but he’s slowly catching up, ”she expressed.
Because Zolgensma is such a new drug, long-term results are unknown, but the parent says she believes this generation of babies with SMA will be the first to reach adulthood.
However, now the family can start 2022 knowing that the little one is receiving the best possible care.
“Last year, we asked ourselves ‘what does the future hold?’, ‘Are we going to lose our baby?’” She said.
“Now there is light, there is a future, and I honestly didn’t think Edward would have a future. I thought his time with us was numbered, but now I don’t believe it at all, “he concluded.
Zolgensma is considered the most expensive drug in the world, although the public health of England assured that it had negotiated an undisclosed discount on its regular price of US $ 2.1 million (1.79 million pounds sterling).
Novartis, a Swiss pharmaceutical company, obtained permission to market in Russia the drug Zolgensma abeparvovec, the most expensive in the world, which is used to treat spinal muscular atrophy (SMA).
On Thursday, December 9, 2021, the Slavic country’s Ministry of Health admitted and registered the drug, the dose of which costs 2,125 million dollars. Spinal muscular atrophy is a very dangerous disease, which condemns those who suffer from it to a shortened life expectancy.
The disease is caused by a mutation in the SMN1 gene, which causes a reduction in the amount of the SMN protein, necessary for the survival of motor neurons.
One of the benefits of the drug, once inside the body, is that it transports the SMN1 transgene to motor neurons and thus causes an increase in the amount of the SMN protein. The drug has side effects, although they are insignificant compared to the symptoms of SMA.
Zolgensma, unlike other medications that must be administered for life, is only injected in a single injection to children under two years of age. Inoculated babies after reaching six months stop losing muscle control, although the drug cannot reverse the damage already inflicted on the body, details the Medical Xpress portal.