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- Drug Development of Valerias Oligonucleotide
The ongoing tests on Valeria's nerve cells regarding changes in electrical voltage and reduction of misinformation have been largely completed and the data analyzed. The drug for Valeria will be manufactured at the beginning of December. But we are not quite there yet, we still need several safety tests until the drug Valeria can be administered. Path of drug development.
Valeria from Horw (Lucerne) suffers from the very rare gene mutation KCNT1. About 100 cases are known worldwide. Due to the gene mutation, Valeria suffers several painful epileptic seizures every day. She cannot develop mentally or motorically.
However, there is a lot of hope for Valeria. Prof. Dr. Len Kaczmarek from Yale University (USA) and a team of neurogenetics are making a drug for Valeria that frees her from cramps and enables her to develop like other children. She will be able to laugh, talk and stand on her feet. The drug is expected to be available to Valeria this year, provided funding is secured.
The development of this unique drug is costly and time-consuming: 1.8 million Swiss francs. Personal savings and generous donations from relatives, friends, acquaintances and strangers have so far contributed half of this sum. 900,000 francs are still missing. Neither health insurance nor IV will contribute to this amount. Therefore, Valeria's family is dependent on YOUR support. Every donation is precious, no matter how small. Together we can do it!
We are a mixed team consisting of family and friends of Valeria. Our aim is to help Valeria with the therapy she so desperately needs. Therefore we have started the crowdfunding campaign "save Valeria". All the work we do is voluntary.
Nothing is impossible! Together we can do it!