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CHLA Experts to Present at 2024 American Society of Hematology Meeting

November 26, 2024

CHLA faculty will share new research on pediatric blood cancers and diseases at the American Society of Hematology Annual Meeting in San Diego.

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.

A young boy with a medium skin tone sitting in a wheelchair and smiling while an adult woman with a medium skin tone steps out of a car and smiles at him, holding his hand.

Research and Breakthroughs

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

July 1, 2024

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.

light skinned child scratching reddened area in inner elbow

Research and Breakthroughs

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.

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Research and Breakthroughs

Five Questions Parents Should Ask Their Child’s Oncologist About Participating in a Clinical Trial

September 19, 2023

Five important questions that parents should ask about participating in a cancer clinical trial.

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

Research and Breakthroughs

Backstory: An 18-Year Journey From Idea to FDA-Approved Therapy

August 15, 2023

A key study led by Dr. David Freyer showed that sodium thiosulfate prevented cisplatin-related hearing loss in children and led to therapy approval.

Advice From Our Experts

Persistent Snoring: Is it Bad?

September 26, 2016

As parents, we never want to admit that our children are any less than perfect. That wasn’t the case with my eldest son, as no morning alarms were necessary in my home. When he was a boy, my son’s

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Research and Breakthroughs

Phase 3 Clinical Trial Shows Benefits of a New Treatment for Children With Congenital Adrenal Hyperplasia

July 22, 2024

In a new paper from Neurocrine Biosciences, CHLA’s Dr. Mimi Kim and collaborators explore using the drug crinecerfont to treat congenital adrenal hyperplasia.

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