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.
From the Nursing Frontline of the Batten Disease Battle
CHOC recently hit its second anniversary of the first Brineura infusion, a novel treatment for CLN2 disease, or late infantile Batten disease.
CHOC at forefront of treating Batten disease
Bringing hope to patients and families, CHOC is among a few hospitals in the country to offer treatment for Late Infantile Batten disease.
