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
‘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
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.
No Longer Night-Blind, Natalia Isn’t Afraid of the Dark
October 19, 2021
After being treated with a breakthrough gene therapy at Children’s Hospital Los Angeles for a genetic eye disorder, 5-year-old Natalia can now see clearly for the first time in her life.
Study: A Novel Medication for the Most Difficult-to-Treat Hemophilia Patients
March 29, 2023
Study results are promising for an investigational drug to prevent bleeding in patients with a severe clotting disorder.