Spread awareness about Batten disease. Fund a gene therapy tiral. Do our share to ultimately find a cure
Dedicated to BEATing Batten disease
Our mission is to help find a cure for Batten disease. We want to do our part and raise enough funding to research and start a clinical trial specifically to halt degeneration in children facing Aashi's type of Batten. Family organizations in the Batten community have been essential in funding research and clinical trials. There are only few families that we are aware in USA who has the same variant as Aashi. We will be joining forces. Our hope is that our efforts will not only help find cure for Batten disease as a whole, but also help combat additional rare diseases affecting hundreds of millions of people worldwide.
Where is your donation going?
All funds will go towards research and a clinical trial for Aashi's variant of Batten. We are in the process of putting together a team of scientists from around the world, working with other similar orgnaizations, with experience in Batten disease and clinical trials. We are focusing on gene therapy as it is the fastest and most cost-effective path to a cure. A gene therapy approach costs roughly $4 million and includes the initial discovery phase (mouse models and safety studies), production of the gene-therapy vector, and ultimately the design and launch of the clinical trial itself. Our goal is to fund research that will help not only Aashi and other children facing her diagnosis, but will also lead to discoveries that could help other diseases.
What is gene therapy?
Gene therapy involves DNA being injected into a patient to treat a genetic disease, such as Batten. The new DNA usually contains a functioning gene to correct the effects of the disease-causing mutation, and is typically introduced via a benign virus. Great strides have been made in gene therapy over the past decade, showing promising results in hundreds of trials. In addition to helping combat Batten disease, gene therapy holds promise for treating a wide range of other diseases, including cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. The more funding that can be provided to gene therapy trials, the closer scientists can get to fixing genetic code mutations and saving millions of lives.
Aashi's Hope Foundation is fundraising through Mila’s Miracle Foundation to Stop Batten. All donations will go through their fiscal sponsor, Creative Visions Foundation, a 501(c)(3) nonprofit organization. All donations are tax–deductible.