MVA Syndrome leaves a handful of children worldwide with serious developmental delay and a very high risk of childhood cancer — and there is no treatment. Founded by a family living with MVA, we’re funding the search for the first one: testing approved medicines that could reach these children fast.
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Mosaic Variegated Aneuploidy (MVA) is an ultra-rare genetic condition affecting only a handful of children worldwide, including the child whose family founded this charity. It causes growth problems, developmental delay and a very high risk of childhood cancer. At its heart is the loss of a protein called BubR1, which helps cells divide safely. Today there is no treatment for the condition itself; families are left with only supportive care while their children face life-limiting complications.
In 2027 we will fund the creation of a ‘drug screen’ - a systematic way of testing existing, approved medicines and nutritional supplements to find those that can restore BubR1 and correct the biology driving MVA. By focusing on treatments that already exist, we can move promising candidates towards children far faster and more affordably than developing a new drug from scratch. Our aim is to identify one or more drugs ready to progress towards treatment.