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Life With Hemophilia, Reimagined: Ryan's Gene Therapy Journey

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After receiving Hemgenix gene therapy at Children's Hospital Los Angeles, the 24-year-old is managing hemophilia B with no more infusions—and a newfound freedom.

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“Meaningful Hope:” Elevidys Gene Therapy Helps Eliot Move With Ease

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At 4 years old, Eliot was the first patient at CHLA to receive Elevidys, a groundbreaking new gene therapy for Duchenne muscular dystrophy. The treatment has been a game changer for his health.

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Elevidys Gene Therapy: A Neurologist’s Perspective

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A CHLA expert shares her experience of administering this gene therapy for Duchenne—and why comprehensive care is critical for patients.

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100 Vision-Saving Procedures and Counting: Q&A With Retinal Gene Therapy Expert Dr. Aaron Nagiel 

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A national leader in pediatric gene therapy, CHLA has treated more patients with the sight-saving Luxturna than any other center in the country.

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Under the Microscope: Unlocking the Mystery of Down Syndrome Regression Disorder

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Jonathan Santoro, MD, is pioneering a novel treatment—and the first clinical trial— for Down syndrome regression disorder, a devastating condition in young people with Down syndrome.

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Gene Therapy Saves Greigh From a Paralyzing Muscle Disease

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Newborn screening found that Greigh had spinal muscular atrophy. He was sent to Children’s Hospital Los Angeles, where gene therapy saved him from paralysis.

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Childhood Leukemia Isn’t What It Used to Be: Latest Treatments, Research, and Reasons to Hope

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Facing a new diagnosis? A pediatric oncologist explains what the care journey looks like for acute lymphoblastic leukemia, the most common form of childhood cancer.

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