CHOC joins drug trial for rare disease that devastates families
CHOC Children’s are participating in a clinical trial of a drug intended to treat a rare pediatric disease called MPS IIIA
CHOC Resident Follows in His Father’s Footsteps
CHOC performs first in-human gene therapy dosing for MPS I
Physicians at CHOC Children’s Hospital recently administered the first ever in-human dosing of gene therapy for Hurler Syndrome, a rare and progressive lysosomal storage disease.
Journey to a rare diagnosis: Colten’s story
A CHOC PICU nurse tells the story of her son, Colten, who received a rare Alström Syndrome diagnosis with the help of specialists at CHOC Children's.
