Two children from a Brooklyn family were born with an ultra rare genetic disease called Canavan disease. Their parents’ research revealed that gene therapy could possibly treat the disease. Unfortunately, they will have to fund the gene therapy treatment themselves. They set up a GoFundMe account to raise money and have contacted gene therapy companies and specialists. Canavan disease is a fatal inherited genetic neurological disease that causes increased head circumference, lack of head control, muscle stiffness or muscle floppiness, seizures, blindness, trouble swallowing and deafness. New research in gene therapy has shown promising results to treat many rare genetic diseases, but the cost of this treatment is a huge economic concern.
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