Our Patients

One family shares their journey with the groundbreaking gene therapy Luxturna, which helps restore vision loss caused by a rare retinal disease.

An infant’s successful liver transplant demonstrates the viability of organ donations from older, living adults.

Born with Pierre Robin sequence and hospitalized her first three months of life, Julie Rose, now 8, greets each day with positivity and confidence.

CHLA’s Substance Use Prevention and Treatment team uses an age-old Indigenous ritual to support its young patients in their journey toward recovery.

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.

The successful outcome of Henry’s case proved to be a turning point in the hospital’s move toward performing cleft repairs on younger babies.

The risky decision to use a blood thinner to shrink the large clot, which took up nearly all of Birdie’s right atrium, turned out to be a lifesaver.

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.

A medical odyssey with epilepsy led Lucia’s parents to CHLA, where they found the diet therapy treatment that Lucia’s brain needs to thrive.

Born with grave cardiac defects, Aaliyah was dying. A CHLA heart surgeon devised a way to keep her alive for close to 500 days until a donor heart was found.