CHOC is one of five Batten Disease Centers of Excellence across the country and the only one on the West Coast.
Improving outcomes and treatments for lysosomal storage disorders
Learn how the CHOC Foundation of Caring Lysosomal Storage Disorder Program and Metabolic Lab are advancing lysosomal storage disorder treatment.
CHOC receives prestigious NIH grant to study treatment for rare lysosomal storage disease
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.
