CHOC selected as West Coast site for gene therapy clinical trial for patients 8 years and older with Glycogen Storage Disease type 1a.
Dr. Jose Abdenur
At the inaugural CHOC/UCI Rare Disease Conference, providers discussed plans for extended research, collaboration and treatment strategies.
The Inaugural CHOC and UCI Rare Disease Symposium & Family Conference will take place on Friday, March 11, 2022, from 10:30 a.m. to 3 p.m.
CHOC and UCI Health’s new designation is expected to attract more resources and recognition to help find novel cures for rare disorders.
Families with kids who have rare metabolic disorders are coming from all over the U.S. for investigational gene therapy treatments at CHOC.
Using machine learning algorithms and artificial intelligence could make a dramatic difference in diagnosing children with rare diseases.