Our beloved daughter 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). We were very happy to have her and we were observing her new movements and her development day by day. In December, we realized some weaknesses in her movements. When we consulted to the doctors, they said that it could be because of her low weight. Later, doctors said that the problem resulted from muscle weakness and she needed rehabilitation. Then we went to the rehab center. In January, her condition got worse and we took her to the hospital to have her blood tested.
She was diagnosed with SMA TYPE1 which is a rare genetic disease caused by a mutation in the survival motor neuron1(SMN1) gene. When we learned her disease,we got devastated. And now our one and only desire is to fight against this and make her struggle for life.
This disease prevents the SMN1 gene to produce the necessary protein which helps the movements of muscles. As a result of this inefficacy of the functional SMN1 protein, the motor neurons die, leading to debiliating and often fatal muscle weakness. Children with this condition have difficulties in holding their heads up, swallowing and breathing. And mostly they don’t survive due to respiratory failure.
Mia seems like a normal baby, she is full of life and likes to smile.
She takes rehab everyday, however it isn’t enough, it doesn’t help her get over this disease.
In Poland, the government supplies Spinraza (the injection supporting the protein produced by SMN2 gene in the body). With the help of Spinraza, the SMN2 is supported but it doesn’t help SMN2 gene to produce enough protein to make her body move well. Unfortuantely, the injection has no impact on the production of SMN1 gene. Spinraza 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 us most. If Spinraza is taken out of the medical list, we can’t afford it because each dose costs 90 thousand Euros. When Spinraza is injected to a child’s body, it doesn’t mean that it operates the same in every child. So, the results aren’t predictable.
Our daughter Mia’s only chance is to take ZOLGENSMA.It costs 2 MILLION DOLLARS. This price makes this medicine the world’s most expensive treatment. Zolgensma 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.
Our daughter means everything to us and we don’t want her die suffering day by day from this disease. We want to have this gene therapy but we can’t afford it by no means.
We are begging the kindhearted people to help us to save Mia’s life!
Please give us a chance for getting her healthy life back. We are running out of the time, so we need your urgent help.