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.
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.
How CHOC otolaryngologists are changing tonsillectomy recovery
Two CHOC otolaryngologists are championing intracapsular tonsillectomies to reduce bleeding and hospital readmission rates.
