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.
Rare mitochondrial diseases are the focus of Feb. 10 virtual CureARS event
The upcoming webinar is open to the public, and will share the latest developments in the battle against rare mtARS mitochondrial diseases.
CHOC conducts clinical trial for Glycogen Storage Disease type 1a
CHOC selected as West Coast site for gene therapy clinical trial for patients 8 years and older with Glycogen Storage Disease type 1a.
