Content related to What Is Gene Therapy?

Gene therapy holds tremendous potential for children, but many promising therapies are shelved. Here’s what a new model could look like.

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.

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.

Routine newborn screening can reveal genetic disorders early to enable early interventions that help children and save lives.

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.

The new approval opens the gene therapy to a wider range of children with Duchenne muscular dystrophy, a rare genetic disorder that worsens over time.

Four investigators share their research—and how the USC/CHLA facility will help bring novel cell therapies to clinical trials faster.

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