CHOC and UCI Health’s new designation is expected to attract more resources and recognition to help find novel cures for rare disorders.
Metabolic disorder families increasingly turning to CHOC for historic gene therapy treatments
Families with kids who have rare metabolic disorders are coming from all over the U.S. for investigational gene therapy treatments at CHOC.
Batten disease patients highlight CHOC’s growing reputation as a destination for kids with rare conditions
Children from throughout the U.S. travel to receive CLN2 treatment at CHOC, the largest Brineura infusion center in the nation.
CHOC joins drug trial for rare disease that devastates families
Dr. Wang and his team at CHOC are participating in a clinical trial of a drug intended to treat a rare pediatric disease called MPS IIIA.
CHOC receives $8 million to advance research for rare disorder
An $8 million gift from the Foundation of Caring will help CHOC advance research for a rare lysosomal storage disorder, Pompe Disease.
CHOC performs first in-human gene therapy dosing for MPS I
CHOC clinicians recently administered the first ever in-human dosing of gene therapy for Hurler Syndrome, a rare lysosomal storage disease.
