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Valeria Schenkel was born on 14 February 2018 in Switzerland. A few days after her birth, the little girl suffered her first epileptic seizure. The resulting diagnosis is devastating. Due to a de novo mutation on the KCNT1 gene, Valeria would be denied any cognitive or motor development in her life. Since then several seizures occured daily and Valeria suffered every day. Valeria's parents, Alexandra and Mario Schenkel fought for their daughter. Together with Yale School of Medicine and neurogeneticsts from Harvard Medical School (Boston Children's Hospital), they have set up a program that corrects the de novo mutation on the KCNT1 gene and stops its fatal effects. An Antisense Oligonucleotide named after Valeria - "Valeriasen" was first administered in September 2020. Valeria's seizures declined drastically but unfortunately Valeria lost her fight against her devastating disease in September 2021. Valeria has achieved many things in her short life. She has demonstrated, that drugs for orphan diseases can be developed in record time. Dosed correctly and given to other children with proper precautions and regular monitoring, Valeriasen is a promising drug. Other children are already being treated with it. Valeria has paid a very high price, but in return she has brought science a decisive step forward. Thanks to her fight for a chance in life, many children with a rare disease will be able to live a healthier and happier life.