Discover how Rady Children’s Health Orange County is studying gene therapy as a less burdensome treatment alternative for children with Hurler syndrome.
From unanswered questions to transformative care: 2026 Rare Disease Symposium and Family Conference
The gathering brings together clinicians, researchers, patients, families, and industry partners — fostering awareness, equity, and progress in rare disease diagnosis and treatment.
Research Day caps historic year after institute’s expansion into new state-of-the-art home
“Every question we ask, every study we undertake, and every breakthrough we pursue begins with a single patient – the child whose story sparks curiosity, inspires discovery, and drives progress.”
CHOC earns national distinction as Batten Disease Center of Excellence
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).
