I am Anastasia Darbinyan, the mother of three daughters – Elina, Milla and Sofia. My youngest daughter Sofia has been diagnosed with a rare genetic disease – spinal muscular atrophy. This disease gradually takes away the ability to move, breathe, swallow, the heart to beat, and finally, leads to death. Moreover, this is a very rapidly progressing disease, and kids with such a diagnoses are classified as disabled.
Currently Sofia is 4 months old but she is not able to hold her head and turn her body. She is falling behind in her motor development – level of 0-1 month age. However, psychologically she is absolutely fine – she smiles, laughs and responds to interactions.
There are two ways of treatment that exist in the world – lifelong treatment and a one-time treatment. Both are extremely expensive and we cannot afford it. We have tried all possible options and exhausted resources and opportunities that we had.
There is one medicine that is available in Russia – SPINRAZA (Nusinersen), a drug that has to be taken throughout ones life. Unfortunately, our insurance does not cover it. Moreover, we are eligible for getting help from the Russian government that is supposed to provide us with this drug but due to its limited budget they are stretching the time by complicating the process with bureaucracy. While we are fighting for our rights to get the treatment our daughter loses motor neurons and her muscles get atrophy. Thus there are big chances that we will not be getting the treatment regularly and in time, so we lost hope and are fearful to lose our daughter.
Thanks to gene engineering there is one more drug that can help, which has been developed recently and registered in the USA and Europe - ZOLGENSMA. This injection is given only once and, for the full success and recovery, should be given until the age of 6 month old. In this case there are chances for recovering of motor development and getting back full ability to move and live a regular life, according to the tests and studies.
We have created a fund to collect money for Zolgensma, and we are asking for your help. Please help us to save our daughter’s life.
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