We have successfully raised over 1 Million Dollars! and as a result, we now have contracts with Sick Kids Hospital and UMASS Chan Medical School for the Research of the variants to RBM28 causing disease and the Development of Gene Therapy for Lucas' variant to RBM28.

Both Sick Kids Hospital and UMASS Chan now offically have mice born. This is a huge step in the research required both to understand the disease and develop the Gene Therapy because the loss of function to RBM28 is lethal meaning that without this gene, mice (and humans) will not be born. 

Both research teams will now breed these mice multiple more times until they can successfully genotype the mice to have both RBM28 gene's with the variant causing the disease. Behavioural studies will also be able to show if the mice shows symptoms of the disease the same as it does in a human. 

Following genotyping and behavior studies, Vectors (gene therapy) will be delivered to the animals and more behaviour studies will continue to analyze symptoms. 

Without the success from fundraisers and the support of the community. None of this would be possible. We are afforded the luxury for research to have started so quickly because to date, funding has been available for this first phase. 
We continue to fundraise so that we can continue to fund this reasearch and development to the Clinical Trial.