A recently completed pilot program at CHOC and other California clinical sites helped target a baby’s specific genetic disease in just days.
CHOC earns $1 million grant to advance rare disease genome-editing therapy
CHOC will advance rare disease genome editing therapeutics, thanks to a $1 million grant from The Larry and Helen Hoag Foundation.
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.
Offering answers, hope through rapid whole genome sequencing
Project Baby Bear is a pilot project, including CHOC and four other children’s hospitals, to use rWGS to save babies’ lives.
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.
