Written by Holly Alyssa MacCormick South San Francisco, CA, January 30, 2026 โ As many as 1 in 10 people in the U.S. has a rare disease, many of which have no known cure. A new program by the California Institute for Regenerative Medicine (CIRM) could change this by funding novel ways to develop multiple … Continue reading CIRM board supports a rare disease funding plan, administrative changes
Rare diseaase
Early-stage research could benefit those with genetic diseases
Written by Holly Alyssa MacCormick Image Credit: Audrey Davidow๏ผCalvin outside Wanting the best for your child is a universal experience, and Audrey Davidow wanted her baby to have a bright future full of possibility. But in March of 2012, her 13-month-old son Calvin was diagnosed with a rare disease called Pitt Hopkins Syndrome, and she … Continue reading Early-stage research could benefit those with genetic diseases
Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment
A 12 year-old Kurt Gillenberg. Photo courtesy of Kurt Gillenberg. When Kurt Gillenberg was 10 months old, his parents knew something wasnโt right. But it wasnโt until he reached 18 months that they found a doctor at the University of California, San Diego, who diagnosed Kurt with cystinosis โ a rare genetic, metabolic defect that … Continue reading Breaking barriers in rare disease: A gene therapy transforms cystinosis treatment
How CIRM-funded research is bringing a lifesaving gene therapy to a 5-year-old
For her first year of life, Seersha Sulack stayed mostly in her bedroom because something as simple as a common cold could have killed her. The five-year-old was born with ADA-SCID, a condition so rare that only eight babies a year are born with it in the United States. Now, thanks to the work of … Continue reading How CIRM-funded research is bringing a lifesaving gene therapy to a 5-year-old
Bridging the divide: stem cell students helping families with rare diseases become partners in research
CIRM's Bridges students and Rare Science's families with rare diseases Sometimes itโs the simplest things that make the biggest impact. For example, introducing a scientist to a patient can help them drive stem cell research forward faster than either one could do on their own. Want proof? This year, students in CIRMโs Bridges to Stem … Continue reading Bridging the divide: stem cell students helping families with rare diseases become partners in research