Grant’s support will help advance gene-therapy treatment for the rare lysosomal storage disease mucopolysaccharidosis type I (MPS I).
Enzyme replacement therapy slows progression of CLN2 Disease
CHOC is at the forefront to offer breakthrough enzyme replacement therapy to improve the quality of life for children with CLN2.
CHOC joins select group of U.S. healthcare systems designated as a Rare Disease Center of Excellence
CHOC and UCI Health’s new designation is expected to attract more resources and recognition to help find novel cures for rare disorders.
Metabolic disorder families increasingly turning to CHOC for historic gene therapy treatments
Families with kids who have rare metabolic disorders are coming from all over the U.S. for investigational gene therapy treatments at CHOC.
Batten disease patients highlight CHOC’s growing reputation as a destination for kids with rare conditions
Children from throughout the U.S. travel to receive CLN2 treatment at CHOC, the largest Brineura infusion center in the nation.
CHOC joins drug trial for rare disease that devastates families
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.
