CHOC clinicians recently administered the first ever in-human dosing of gene therapy for Hurler Syndrome, a rare 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.
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 Researcher Recognized at International Molecular Medicine Conference
In addition to serving as a guest speaker at the conference, Dr. Huang was honored with the “Best Poster” award.
