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.
Research Institute student intern is proving his mettle on neurosurgery team
Student intern with the CHOC Research Institute gets valuable experience from working on research studies and shadowing surgeons.
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).
‘We will have a better tomorrow for these kids’
Parent provides powerful testimony at second-annual CHOC and UCI Rare Disease Symposium & Family Conference
Second-annual CHOC-UCI Rare Diseases Symposium set for March 10, 2023
At the annual symposium, hundreds will gather to share about advances in rare disease research and a unifying theme: hope.
