Our mission is to fund the gene therapy work at UTSW and Children’s hospitals in Dallas, and to help other families with children diagnosed with rare neuro-degenerative diseases become their own advocates for gene therapy. We are working with other non-profit organizations, scientists, and doctors from UTSW and Children’s who are dedicated to helping children like Joseph get the treatment they need to stop the progression of the disease.
Our goal is to raise $1.2 million for a new clinical trial to give children like Joseph the chance to stop their disease, and to deliver hope to families confronted with similar diagnoses.
Your help is pivotal in raising the necessary funds and awareness. Every dollar and effort makes a difference in getting closer to our goal–we cannot do this without you. Become a Friend of Joseph now, and join us in changing the world for children and families through gene therapy.