Student intern with the CHOC Research Institute gets valuable experience from working on research studies and shadowing surgeons.
Grant’s support will help advance gene-therapy treatment for the rare lysosomal storage disease mucopolysaccharidosis type I (MPS I).
Parent provides powerful testimony at second-annual CHOC and UCI Rare Disease Symposium & Family Conference
At the annual symposium, hundreds will gather to share about advances in rare disease research and a unifying theme: hope.
The upcoming webinar is open to the public, and will share the latest developments in the battle against rare mtARS mitochondrial diseases.
CHOC selected as West Coast site for gene therapy clinical trial for patients 8 years and older with Glycogen Storage Disease type 1a.