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Four families struggle to finance SPG50 gene therapy for their children

Four families struggle to finance SPG50 gene therapy for their children

Families Raise Funds to Treat Rare Genetic Disorder Four American families are urgently trying to raise $1.15 million to save their children from a devastating genetic disease. While treatments are available, the financial burden falls on them to fund clinical trials. Each family has a child diagnosed with spastic paraplegia 50 (SPG50), a rare neurological […]