Our little family member Ksenia (4.5 months old) is sick with a deadly genetic disease - Spinal Muscular Atrophy type 1. To safe her life we need to enroll her to clinical trial which is not available in Russia.
We are grateful for any possible support!!
Hello, i am Maria. I am the aunt and godmother of this little cutie named Ksenia. We are from N.Novgorod, Russia.
It was a complete shock to hear her diagnosis. Kseniia is sick with a deadly disease Spinal Muscular Atrophy (SMA) type 1. This is very severe type, doctors told us she may live few months more, but no longer than year or two, if we don't do anything about it. There are examples of some "parents" who just leave things so and leave their child to die but, what to say - even animals don't leave its generation like that. We believe she can and will survive!!
At this moment she is not able to move her legs, yet she shows movement on hands and has some movement to turn head. Though SMA is a genetic disease and it is progressing, her lungs are not developing, and her muscles are getting more and more disabled.
Here you can hear from Russian doctors just "prepare for funeral".
But, in our speedy progressed World we already have Spinraza - first in the World medication to treat SMA. It was approved by FDA at the end of 2016, though it is not possible to be accessible for foreigners. There are also clinical trials aim to completely reverse SMA. Just in Russia we don't have it.
This is why we need to go and give chance for our little family member Ksenia to be enrolled to one of this clinical trial's program - the company Roche is launching a clinical trial, just 40 babies for whole Europe to have a chance for life, chance to survive!! And we believe that miracles will happen for our Ksenia!!
Ksenia was seen by doctor in Belgium at 20th December 2016, and she can be enrolled to trial which can safe her life!
We are going there at the begging of February 2017 to enroll her IN the clinical trial which will safe her life!
We may provide any information that you need in case you wish to help us. We are all under one sky, even a moral support means a lot for us!
We are not a rich family, but still we are doing all will do all possible to safe her life. This is all very expensive for us, to take care of a disabled child, to buy all needed equipment and devices for her (such as BiPap, artificial lung ventilation facility, cough assistant, etc.), travels, documents preparation and other, our budget is getting low fast, its very hard to stay strong. At this hardest moment for our family yet we have a huge hope and prayers for miracles to happen.
We need any possible support and we believe in good people in the World. Ksenia can have a chance for life, and we will do all for it. This little cutie wants to survive so much, just look in her eyes and you can see it!
We are grateful for any your support, even moral support is highly important for us.
Thank you so much for understanding and supporting us!
I will give all contacts in case you need more information (WhatsApp, Facebook, Skype, Viber, etc)