SMA: A treatment is there, but not for everyone

Why aren’t children and adults with rare disease getting access to treatment, this is a good question. Miles was diagnosed with the disease when he was just 13 months old. His parents knew that something was not commonplace, because he had not started to crawl and seemed to be weaker than any other child. Without treatment, the course of SMA is a progressive deterioration of motor neurons. Mobility and motor function are lost.

SMA is the number one genetic killer in infants under two year

Progress in research

Researchers knew for more than a decade that SMA was caused by a genetic mutation of the SMN-1 gene. They also knew that there was a second, inefficient partner gene called SMN-2 that was almost identical.

The breakthrough in the treatment of SMA came in 2016.
Spinraza from Biogen was launched on the marked. A drug that successfully turn SMN-2 into a working version of SMN-1.

So far, there was no drug that could slow the disease.

Nevertheless, the therapy is not available to everyone

Countries like Canada, Switzerland, Finland, Denmark are fighting for access to treatment

Germany has exemplary access to treatment
Adults, children have access to the Spinraza treatment. There is no age limit or discrimination due to age

This is SMA, this is Spinraza treatment

Mia

Mia

Mother of three childrens. One of the twins my eldest son had SMA Typ II -III. He passad away and spread his wings into the sun . He lived 3 years and seven months. Fly my sweet little honeybee🧚‍♂️🦋 fly my little Honeybee 🐝

4 reaktioner till “SMA: A treatment is there, but not for everyone

  • 18. mars 2018 kl. 14:09
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    Excellent post Dude 🙂

  • Mia
    19. mars 2018 kl. 11:09
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    ❤️

  • 19. mars 2018 kl. 19:13
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    Mia-Dein Mann ist ein Künstler im schreiben, ich lausche gerne seinen Worten. 🙂

  • 23. april 2018 kl. 10:33
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    We have a rare disease and we’re not being heard and we’re not getting the treatment we deserve

    Why 2 Calgary women are lobbying governments to cover costs of the life-saving drug, Spinraza. Last June, Health Canada approved the new drug because it can lessen symptoms and can potentially improve quality of life. It is the only approved treatment for SMA available.

    According to the maker of Spinraza, the first year of treatment costs $700,000 CAD with every year after that costing $350,000 CAD

    Read more

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