CHOC is at the forefront to offer breakthrough enzyme replacement therapy to improve the quality of life for children with CLN2.
Children from throughout the U.S. travel to receive CLN2 treatment at CHOC, the largest Brineura infusion center in the nation.
CHOC is one of the state’s largest referral centers for the newborn screening program, which checks babies for many serious genetic disorders.
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.
CHOC recently hit its second anniversary of the first Brineura infusion, a novel treatment for CLN2 disease, or late infantile Batten disease.