Natural History Study
Tomorrow we are flying to Minnesota to participate in a Natural History Study for children with Sanfilippo type A. It is exciting to know that researchers are actively working to find a treatment option and hopefully a cure for these kids. Shire Pharmaceuticals will gather information on the children throughout 3 full days of testing, and eventually ERT (enzyme replacement therapy) will go to trial in the US. I am not sure if Waverly & Oliver will benefit from ERT or other forms of treatment, due to their age and the progression of the disease. However, it feels great knowing that the information they provide is going to help other families in the future. Maybe one day when a family is told their child has Sanfilippo, they will also be told that there is a treatment available and their child will live a long, happy life. That is why we are taking part.