US$103.00raised of $12,000,000.00 goal goal
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Please help save Arthur. He needs the most expensive medicine in the world ($2,125 million dollar)
Arthur this lovely baby boy in this picture, from Osasco – Brazil, he has SMA type 1, a terrible, degenerative disease, that is slowly taking Arthur from us.
He fights for living since he was 6 months old, when he was diagnosed with: SMA (Spinal Muscular Atrophy) type 1 (the most severe). SMA is a rare genetic disease, characterized by weakness and muscular atrophy, followed by fasciculations severely compromising mobility during an early stage. Without proper treatment, the life expectancy of a child with SMA type 1 is 2 years old, but we change that!
The medicine that can stop the disease is called Zolgensma. The cost, 2,1 million (USD), or, R$11 million (BRL) approximately, just the medicine, Zolgensma. Hospitalization costs and exams are still to be priced and expected to be expensive. We don’t have much time left, because the treatment can only be applied to children within 02 years of age because it is a gene therapy, we only have a few months left to raise the funds for the treatment.
On May 24th, 2019 the FDA approved Zolgensma in the US, from AveXis (pharmaceutical company Novartis) that promised better prognosis and improved results, being capable of even stopping the disease because of its different action mechanisms. The new medication, Zolgensma, promotes the genetic material’s transference by a vector (virus), correcting the altered DNA and not by just replacing bases and raising SMN2. By doing that, roughly speaking, a “genetic material transplant” occurs, stopping in a definitive way the disease with only one intravenously dose.
Arthur has been taking Spinraza (Nusinersen), with a prescription for 06 doses during the first year of life and 03 more annual doses for the rest of her life. Although he has taken 1 dose, he hasn’t presented significant signs of improvement.
To explain this difference between Spinraza and Zolgensma, we must clarify that Spinraza acts only over the gene SMN2, raising the protein SMN’s “productivity”. However, the gene responsible for the main motor neuron is the SMN1, which is absent or defective in cases of patients with SMA.
Therefore, this treatment is our one and only hope to save Arthur.
We kindly ask for your donation and engagement on social media to help us raise funds to have the treatment.
Help us save Arthur! What if it was your child?
- Arthur Sales
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