A couple of Chicoutimi, ready to do anything to save his little Nathan, embarks on the quest crazy to raise$ 2.8 Million to offer his boy the only treatment that can help, is the Zolgensma, ” medicine is the most expensive in the world “.
Barely two months after his birth, the little Nathan Ouellet was diagnosed with a rare genetic disease, spinal muscular atrophy type 1.
Caused by a gene missing, the disease atrophy gradually the muscles of the body, ultimately resulting in respiratory distress or a problem of swallowing which can lead unfortunately to the death.
Nathan has started a process from which helps to fend off the effects of the disease, but the latter will never make gains.
And despite this invasive treatment that requires three lumbar punctures per year, there is no guarantee that it can one day stand or even just hold her head.
The most expensive in the world
This is where the famous Zolgensma. Still not approved by Health Canada, the drug showed impressive results elsewhere in the world.
The catch ? It is the most expensive in the world, to$ 2.8 Million for the single injection. And all this must be done before the child reaches two years, by October 2021.
“It is as if we said to you that something can save your son, but that is completely unattainable “, tell us with dismay his parents, Marc-André Ouellet and Genevieve Side.
The couple, however, opted to roll up the sleeves and tackle the huge mountain which separates them from the treatment.
“We will do everything for him, even if that means having to achieve the impossible “, they say.
The family turned to the sociofinancement since there is no other realistic way to raise such a sum by themselves.
Even more on a daily basis, they need to manage follow-ups and exercises which are vital to Nathan, in addition to seeing the development of his twin brother, Jake, who is not suffering from any health problem, fortunately.
“This is heartbreaking because it makes us feel that the life of our sons has a price and that this price, it is$ 2.8 Million,” sighs Marc-André Ouellet.
Sense of urgency
Launched Thursday evening, the campaign sociofinancement who will take on the allure of a marathon has already raised about $35,000.
A first pat on the back for parents who hope that their touching request we will respond.
“There is a sense of urgency because the treatment must occur before age two, but there is also a sense of urgency because every second that passes, Nathan loses the capabilities that he does, she will regain never. It must go as fast as possible, ” says Geneviève Caron, in a real cry from the heart.
“Like anyone, we want what is best for our children, and in this case it is the Zolgensma. We have seen children who have received the treatment, starting to walk, that the medicine had never seen. “
THE DRUG ZOLGENSMA
- Gene therapy used for spinal muscular atrophy type 1
- Developed by the american company AveXis, bought by Novartis
- Approved in the United States in may 2019 and in Europe in may 2020
- Still not approved by Health Canada
- Its cost of$ 2.8 MILLION CA in fact the medicine is the most expensive in the world
WHAT IS SPINAL muscular ATROPHY TYPE 1
- Neurodegenerative disease genetic
- Prevalence approximately 1 in 10,000 births
- Due to atrophy of the muscles of the body
- Without treatment, few children survive beyond two years
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