Mia Medical Costs SMA

Please read this story:

Mia SMA

Mia was born in the year of 2019 in July. When she was born she was only 2500 gr.(she weighted less when compared to other newborns).

She was diagnosed with SMA TYPE1 which is a rare genetic disease caused by a mutation in the survival motor neuron1(SMN1) gene.

Treatment in Poland will be injected to Mia’s spinal cord in every four month for a lifetime. And this is supported by the government only for next year. When the government stops the support, what will happen? We don’t know either and this is what scares most.

Mia’s only chance is to take Gene therapy. It costs 2 MILLION DOLLARS. This price makes this medicine the world’s most expensive treatment. Gene therapy virus work very similar to SMN1 gene and allows it to produce the necessary protein which is critical to the function of the nerves that control our muscles. This gene therapy is given as a one time infusion into the vein. The only condition to have this therapy is to be less than 2 years-old.

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