CHILDREN'S HOSPITAL LOS ANGELES

Children's Hospital Los Angeles researchers have become the first in the world to investigate using genetically engineered pig hearts as a "bridge" for infants awaiting heart transplants.

The FDA has approved trofinetide (developed by ACADIA Pharmaceuticals) as the first-ever treatment for Rett syndrome, a rare genetic disorder primarily affecting females that causes severe developmental delays and neurological symptoms.

Children's Hospital Los Angeles (CHLA) celebrates administering its 100th retinal gene therapy, Luxturna, since 2018 for Leber congenital amaurosis (LCA).

A new study at Children's Hospital Los Angeles reveals that magnetic resonance elastography (MRE) and transient elastography (TE) demonstrate insufficient accuracy in detecting high-grade liver fibrosis in pediatric MASLD patients.