Four families are desperate to raise funds for their children’s paralyzing disease

Four American families are rallying together to raise $1.15 million by October for a crucial phase 3 gene therapy trial targeting SPG50, a rare genetic disease affecting their children. This effort highlights the urgent need for funding in rare disease research and the hope that gene therapy can provide a breakthrough for those affected. Their determination not only showcases the strength of community support but also emphasizes the importance of advancing medical treatments for rare conditions.