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 Diseases
Seeing is believing: how some scientists – including two funded by CIRM – are working to help the blind see
“A pale hue”. For most of us that is a simple description, an observation about color. For Kristin Macdonald it’s a glimpse of the future. In some ways it’s a miracle. Kristin lost her sight to retinitis pigmentosa (RP). For many years she was virtually blind. But now, thanks to a clinical trial funded by … Continue reading Seeing is believing: how some scientists – including two funded by CIRM – are working to help the blind see
How the Ice Bucket Challenge changed the fight against ALS
200 people in Boston take the Ice Bucket Challenge: Photo courtesy Forbes A couple of years ago millions of people did something they probably never thought they would: they dumped a bucket of ice cold water on their head to raise awareness about a disease most of them had probably never heard of, and almost … Continue reading How the Ice Bucket Challenge changed the fight against ALS
CIRM-funded stem cell clinical trial for retinitis pigmentosa focuses on next stage
How retinitis pigmentosa erodes normal vision The failure rate for clinical trials is depressingly high. A study from Tufts University in 2010 found that for small molecules – the substances that make up more than 90 percent of the drugs on the market today – the odds of getting from a Phase 1 trial to … Continue reading CIRM-funded stem cell clinical trial for retinitis pigmentosa focuses on next stage
A Dream made me change my mind. Almost.
Dream Alliance: photo courtesy Daily Telegraph, UK On Friday I was faced with the real possibility that a horse had made an ass out of me. Over the years we have written many articles about the risks of unproven stem cell therapies, treatments that have not yet been shown in clinical trials to be safe … Continue reading A Dream made me change my mind. Almost.
Patients are the Heroes at the CIRM Alpha Stem Cell Clinics Symposium
UCSD’s Catriona Jamieson and patient advocate Sandra Dillon at the CIRM Alpha Clinic Network Symposium Sometimes, when you take a moment to stand back and look at what you have accomplished, you can surprise yourself at how far you have come, and how much you have done in a short space of time. Take the … Continue reading Patients are the Heroes at the CIRM Alpha Stem Cell Clinics Symposium
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
Rare Disease Day, a chance to raise awareness and hope.
Battling a deadly disease like cancer or Alzheimer’s is difficult; but battling a rare and deadly disease is doubly so. At least with common diseases there is a lot of research seeking to develop new treatments. With rare diseases there is often very little research, and so there are fewer options for treatment. Even just … Continue reading Rare Disease Day, a chance to raise awareness and hope.
The Stem Cellar 