Tomorrow, February 28th, is Rare Disease Day. It’s a day to remind ourselves of the millions of people, and their families, struggling with these diseases. These conditions are also called orphan diseases because, in many cases, drug companies were not interested in adopting them to develop treatments. Here at the California Institute for Regenerative Medicine (CIRM), we … Continue reading Join the movement to fight rare diseases
Tay Sachs Disease
Finding a treatment for Tay-Sachs disease
The California Institute for Regenerative Medicine (CIRM) has awarded $4,048,253 to Dr. Joseph Anderson and his team at UC Davis to develop a blood stem cell gene therapy for the treatment of Tay-Sachs disease. Tay-Sachs disease is a rare genetic disorder where a deficiency in the Hex A gene results in excessive accumulation of … Continue reading Finding a treatment for Tay-Sachs disease