Children's Hospital Receives $8 Million for Pompe Disease Research

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Children's Hospital of Orange County has announced an $8 million gift from the Foundation of Caring Fund in support of research focused on a rare lysosomal storage disease.

The gift will support a comprehensive, multidisciplinary research program focused on advancing the diagnosis and development of next-generation therapies for Pompe disease, a lysosomal storage disorder in which glycogen builds up in the body's cells and causes life-threatening heart failure and muscle weakness in affected babies.

The program will be led by Raymond Wang, a CHOC metabolic disorders specialist, whose work drew the attention of the Foundation of Caring several years ago when he began treating the great-granddaughter of the foundation's founder for the condition. With previous support from the foundation, Wang and his team are already using CRISPR/Cas9 technology to edit the genome and create animal models of the disease. The latest gift from the foundation will enable the team to expand on that work. In recognition of the gift, the research program will be named the Foundation of Caring Lysosomal Storage Disorder Program at CHOC Children's.

"We are so pleased to support the important work of Dr. Wang and his team at CHOC to help find better treatment or, even better, a cure for Pompe disease for patients affected by the condition worldwide," said the foundation's board of directors in a statement.

"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," said Wang. "We're so tremendously grateful to have the Foundation of Caring's support in CHOC's goal to protect the magic of childhood."

(Photo credit: Children's Hospital of Orange County)

"CHOC Receives $8 Million to Advance Research for Rare Disorder." CHOC Children’s Press Release 01/07/2020.