CHOC Receives Grant Toward Rare Infant Disorder

IRVINE, CA — Children's Hospital Orange County received an $8 million gift from the Foundation of Caring to help advance research for a rare disorder that affects infants.

CHOC researchers are working to develop next-generation therapies for Pompe disease, a lysosomal storage disease in which glycogen builds up in the body's cells and causes life-threatening heart failure and muscle weakness in affected babies.

The program, headed by Dr. Raymond Wang, a metabolic disorders specialist, will be named the Foundation of Caring Lysosomal Storage Disorder Program at CHOC Children's, in honor of the gift.

It was Wang's work around Pompe disease drew the attention of the Foundation of Caring several years ago.

Wang began treating the great-granddaughter of the foundation's founder after she was diagnosed with the rare condition.

"This incredibly generous gift from the Foundation of Caring will help accelerate our work to unlock the challenges of Pompe disease and other lysosomal storage disorders, advancing our vision to develop permanent cures for patients with these conditions," Wang said. "We're so tremendously grateful to have the Foundation of Caring's support in CHOC's goal to protect the magic of childhood."