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Batten Disease

Batten Disease is a group of rare, fatal genetic disorders that affect the nervous system. Symptoms typically begin in childhood and include vision loss, seizures, and cognitive decline, ultimately leading to premature death. It is caused by mutations in various genes that impair the body’s ability to clear cellular waste. There is no cure, but experimental treatments like enzyme replacement therapy and gene therapy are being explored. Families often rely on comprehensive care teams to manage symptoms and improve the patient’s quality of life.

Resources

Batten Disease Support and Research Association: https://bdsra.org
NIH Rare Diseases: https://rarediseases.info.nih.gov

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