Part of The Stem Cellar series on ten years of iPS cells For Chris Waters, the motivation behind her move from big pharmaceutical companies and biotech to starting a non-profit organization focused on rare diseases in children is simple: “What’s most important is empowering patient families and helping them accelerate research to the clinical solutions … Continue reading Making a deposit in the Bank: using stem cells from children with rare diseases to find new treatments
rare disease
Patient advocates a small but mighty force at BIO meeting
A few hundred patient advocates operating from a small sub-section carved out of three cavernous exhibit halls could easily get lost amid the 16,000 scientists and business folks attending the BIO International meeting in San Francisco last week. But their voice was heard as they made great use of the meeting to remind companies developing … Continue reading Patient advocates a small but mighty force at BIO meeting
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
The Stem Cellar 