Our association wants to activate and support medical-scientifical research on mutation in the RBCK1 gene and Polyglucosan body myopathy type 1, to develop an effective cure that can eliminate or minimize the damages caused by that alteration.
Our association wants to activate and support medical-scientifical research on mutation in the RBCK1 gene and Polyglucosan body myopathy type 1, to develop an effective cure that can eliminate or minimize the damages caused by that alteration.
My Perfect Mistake Association has the goal to raise funds to activate and finance a new scientifical research, in partnership with Telethon, which is specific on Polyglucosan body myopathy type 1. The research will try to find an effective cure and give hope to children and teenager that suffer from this disease.
Thanks to your donations we can finance the cost for the complete study of Alberto Maria genome to precisely determine which is the clinical picture and how it could evolve. Then we can find a medical procedure to prevent and anticipate every problem caused by mutation in the RBCK1 gene.
My Perfect Mystake Association participate in the Seed Grant program held by Telethon, that has the goal to finance annual research programs. These announcements give the opportunity to create data and new knowledge that will allow to develop new therapeutic approaches.
Understanding the mutation and related developments may be the key to cure children affected by Polyglucosan body myopathy type 1.
