CIRM awarded $4,048,253 to Dr. Joseph Anderson and his UC Davis team to develop a blood stem cell gene therapy that could help people seeking treatment for Tay‑Sachs disease. Tay-Sachs disease is a rare genetic disorder caused by a deficiency in the Hex A enzyme. Without Hex A, certain fats accumulate in brain and nerve … Continue reading Finding a treatment for Tay Sachs disease
Tay Sachs
Rare disease underdogs come out on top at CIRM Board meeting
It seems like an oxymoron but one in ten Americans has a rare disease. With more than 7,000 known rare diseases it’s easy to see how each one could affect thousands of individuals and still be considered a rare or orphan condition. Only 5% of rare diseases have FDA approved therapies People with rare … Continue reading Rare disease underdogs come out on top at CIRM Board meeting