cureFAchildren

We are developing innovative gene therapies to treat Friedreich’s Ataxia (FA), a rare genetic condition, in collaboration with world-leading experts at UTSW. Dr Ryan Butler and Dr. Steven Gray, a pioneer in gene therapy, has already taken eight rare diseases to clinical trials and is helping us develop a breakthrough treatment for FA. Alongside him, Marek Napierala, and Jill Napierala, renowned experts in FA, are working together at UTSW to create life-changing therapies for patients. Our mission is to provide hope and life-changing treatments for those affected by FA, particularly children. We are driven by the desire to save this generation, and we are working tirelessly to bring this treatment to FDA/EMA. We believe in the power of collaboration, and we need your support to make this happen. Your donations will help us accelerate our work and make a real impact. We are determined to save lives and bring relief to families affected by Friedreich’s Ataxia. Together, we can make a difference and create a future where no child has to suffer from this devastating disease.