My name is Alina, and I am Emilia’s mom.

My daughter has an ultra-rare genetic condition caused by a mutation in the QARS1 gene, which affects her brain development and daily life. On the third day after she was born, she started having seizures. Instead of taking our newborn baby home, we found ourselves in the intensive care unit. After a long and painful search for answers, we finally received this diagnosis. We learned that QARS1 is a hereditary disorder caused by mutations in the QARS1 gene, which causes neurodevelopmental delay and frequent, drug-resistant seizures. Today, Emilia cannot sit, walk, or speak. Every day of her life is a fight, for movement, for development, and for the chance to live. We have done everything available to us - therapies, doctors, and constant care, trying to help her. But all of these therapies only manage the symptoms and do not address the root cause of her disease, which will continue to progress.

Now, for the first time, we have a real chance. A chance to develop a personalized therapy that could change her future. We are pursuing the development of a personalized gene therapy that could change Emiliia's future, and those like her. Instead of just treating her symptoms, we are working with experts in this field to develop a gene therapy that can deliver the correct copy of QARS1 to her brain, which should improve the functioning of her brain. This is a complex and very expensive process, and the total cost is approximately $3,000,000. We understand how overwhelming this sounds. However, this process is not impossible - others have developed personalized therapies using similar approaches. We are working with the top experts in this field to develop our therapy and with them, have defined intermediate scientific and funding milestones that will guide our work. We will share our plan and progress with our donors as we continue along this journey. For us, this is the only chance to help our daughter. We are asking for your support. If you are able to donate - thank you from the bottom of our hearts.

If not, please share Emilia’s story, because the right person seeing this can change everything for our daughter.