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.
Content related to As FDA Expands Approval of a New Gene Therapy to Treat Duchenne Muscular Dystrophy, a CHLA Expert Weighs in
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.
What Is Gene Therapy?
August 26, 2024
How next-generation gene therapies target the underlying causes of genetic diseases.
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
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.
‘I Can See! I Can See!’
July 11, 2019
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
Calming the Itch of Liver Disease
December 3, 2024
A new class of drugs can relieve itching for cholestatic childhood liver disease and potentially lengthen time until a liver transplant is necessary.
Trainee Spotlight: Leila Bahmani, PhD
April 21, 2023
Since high school, Leila has been fascinated by how DNA molecules define our fundamental characteristics.
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.