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7 results for "rett syndrome"
Lilly Austyn, 15, attended a special prom in Greensboro with her childhood friend Oliver, creating a milestone her mom feared might never happen. For a teen living with Rett Syndrome, the night proved that inclusion and friendship can turn impossible dreams into reality.
Researchers discovered that different mutations in Rett syndrome require different treatments, opening the door to personalized medicine for children with this developmental disorder. Using advanced brain tissue models, they successfully tested mutation-specific therapies that could help thousands of patients.
Scientists at MIT have discovered what causes leaky blood vessels in children with Rett syndrome and found a way to repair them. The breakthrough points toward potential treatments for a devastating disorder that affects thousands of young girls.
Scientists at MIT discovered why brain blood vessels become leaky in children with Rett syndrome and found a way to potentially fix it. The breakthrough traces the problem to a single overactive molecule that weakens blood vessel walls.
Scientists at Texas Children's Hospital discovered a promising way to treat Rett syndrome, a rare disorder affecting 1 in 10,000 girls. The breakthrough could help 65% of patients by boosting levels of a partially working protein in their brains.
Scientists at Texas Children's Hospital discovered a promising way to treat Rett syndrome, a rare disorder that affects 1 in 10,000 girls and currently has no cure. The breakthrough could help 65% of patients by boosting levels of a partially working protein in their brains.
A 9½-month-old baby became the first person ever to receive a custom gene-editing treatment designed specifically for his unique mutation. The breakthrough offers hope for thousands of rare genetic diseases that were once considered unreachable.