CHOC is at the forefront to offer breakthrough enzyme replacement therapy to improve the quality of life for children with CLN2.
Dr. Raymond Wang
CHOC and UCI Health’s new designation is expected to attract more resources and recognition to help find novel cures for rare disorders.
Families with kids who have rare metabolic disorders are coming from all over the U.S. for investigational gene therapy treatments at CHOC.
Children from throughout the U.S. travel to receive CLN2 treatment at CHOC, the largest Brineura infusion center in the nation.
Dr. Wang and his team at CHOC are participating in a clinical trial of a drug intended to treat a rare pediatric disease called MPS IIIA.
An $8 million gift from the Foundation of Caring will help CHOC advance research for a rare lysosomal storage disorder, Pompe Disease.