Learn how Rady Children’s Health Orange County investigates gene therapy to treat childhood diseases, including Hurler syndrome, liver cancer and more.
‘Something better’? Gene therapy’s potential to treat Hurler syndrome
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.
Parents of 1-year-old with exceedingly rare and life-threatening mitochondrial disease entrust Rady Children’s above anywhere else
With time not on their side, CHOC care team pulls out all stops to save life of Emery Kline.
CureARS and CHOC symposium unites leading rare disease researchers
The event drew participants from representatives of eight children’s hospitals, 15 organizations, and a total of 200 attendees representing 20 states and four countries.
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.
