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A young boy with blonde hair climbs a large tree on a blue-sky day at the park.

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

February 3, 2025

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 female nurse in a blue surgical gown and gloves holds the arm of a young boy with dark curly hair, looking for a vein.

Research and Breakthroughs

Elevidys Gene Therapy: A Neurologist’s Perspective

November 7, 2024

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

Standing infant with dark skin tone and dark hair wearing onesie with colorful dinosaur print smiles as they stand up supporting themselves on a crib railing

Our Patients

Gene Therapy Saves Greigh From a Paralyzing Muscle Disease

September 22, 2023

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.

Our Patients

Ali’s 13,000-Mile Journey of Hope

August 2, 2018

First, he wiggled his fingers. Then he wiggled his toes. Now, he moves his head side to side, and he furrows his eyebrows when he doesn’t agree with you. For 3-year-old Ali Al-Mutawa—and for his

Young boy and mom play with a toy DNA model at the kitchen table.

Research and Breakthroughs

What Is Gene Therapy?

August 26, 2024

How next-generation gene therapies target the underlying causes of genetic diseases.

Our Patients

You will have to grant a parental pardon to Heather for her momentary excessive permissiveness, allowing her 5-year-old daughter to race down the block after dark, free from her mother’s hand. It wasn

Dr. Aaron Nagiel looks into a microscope during bilateral eye surgery for Luxturna gene therapy.

Hospital News

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

November 27, 2024

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

Work That Matters

Trainee Spotlight: Leila Bahmani, PhD

April 21, 2023

Since high school, Leila has been fascinated by how DNA molecules define our fundamental characteristics.

Father with medium skin tone and dark hair smiles as he holds son with medium skin tone and short brown hair and poses with two young daughters in front of decorative altar

Our Patients

Genetic Testing Finds Lethal Heart Disease Lurking in Family

May 3, 2024

CHLA’s Cardiogenomics team cares for three siblings after it was found they inherited a dangerous heart condition called hypertrophic cardiomyopathy.

Content related to As FDA Expands Approval of a New Gene Therapy to Treat Duchenne Muscular Dystrophy, a CHLA Expert Weighs in

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