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One-time gene therapy transforms Sriansh’s future

When Sriansh was just three months old, his family grew concerned as he slept most of the day, arched his back, and could not control his eye movement. His parents brought him to Cincinnati Children’s, where he was diagnosed with AADC deficiency, a rare genetic disorder that disrupts dopamine and serotonin production in the brain. Sriansh’s family was told he might never walk, talk, or lift his head. But thanks to an experimental gene therapy clinical trial, his story took a groundbreaking turn. At just 16 months old, Sriansh became the youngest child in the world to receive a one-time gene therapy treatment, delivered directly into the brain through a minimally invasive surgical technique. His care team inserted an engineered virus carrying the missing gene, allowing his brain to begin producing the essential chemicals it lacked. Today, Sriansh is walking, running, and thriving because of this innovative clinical trial. This pioneering work helped lead to the first-ever FDA-approved gene therapy administered directly to the brain, offering hope for patients with rare genetic diseases once thought untreatable.

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From NICU patient to NICU nurse

Chelsea Fredrickson was born prematurely; at only 26 weeks she was considered a micropreemie. Weighing only one pound nine-point eight ounces, she had to stay in the neonatal intensive care unit (NICU) at Studer Family Children’s Hospital for two months. Today, her journey has come full circle. Wanting to provide the same care that saved her life, Chelsea is a registered nurse in the same NICU, where she cares for some of the smallest and frailest born babies. Like Chelsea’s first few months of life, these children are born extremely early and need extensive care. Every day, she cares for and treats these premature babies, offering them the opportunity for a fulfilling life. Chelsea enjoys connecting with families and sharing her story of being born prematurely, a symbol of hope that every child has a chance at a beautiful life.

Studer Family Children’s Hospital premature birth specialized care