Dr. Stephanie Cherqui, UC San Diego Orphan drug designation is a special status given by the Food and Drug Administration (FDA) for potential treatments of rare diseases that affect fewer than 200,000 in the U.S. This type of status can significantly help advance treatments for rare diseases by providing financial incentives in the form of … Continue reading CIRM-funded treatment for Cystinosis receives orphan drug designation
Dr. Stephanie Cherqui
CIRM Board Approves New Clinical Trial for Rare Childhood Disease
Today the governing Board of the California Institute for Regenerative Medicine (CIRM) approved a grant of almost $12 million to Dr. Stephanie Cherqui at the University of California, San Diego (UCSD) to conduct a clinical trial for treatment of cystinosis. This award brings the total number of CIRM funded clinical trials to 55. Cystinosis is … Continue reading CIRM Board Approves New Clinical Trial for Rare Childhood Disease
The Stem Cellar 