On Rare Disease Day, Emily Walsh, the Community Outreach Director at the Mesothelioma Cancer Alliance, shared how her group is working to raise awareness about rare diseases, as well as the importance funding research new treatments.
SCID
Rare diseases are not so rare
It seems like a contradiction in terms to say that there are nearly 7,000 diseases, affecting 30 million people, that are considered rare in the US. But the definition of a rare disease is one that affects fewer than 200,000 people and the National Institutes of Health’s (NIH) Genetic and Rare Diseases Information Center (GARD) … Continue reading Rare diseases are not so rare
Stem cell heroes: patients who had life-saving, life-changing treatments inspire CIRM Board
It’s not an easy thing to bring an entire Board of Directors to tears, but four extraordinary people and their families managed to do just that at the last CIRM Board meeting of 2016. The four are patients who have undergone life-saving or life-changing stem cell therapies that were funded by our agency. The … Continue reading Stem cell heroes: patients who had life-saving, life-changing treatments inspire CIRM Board
Stem cell stories that caught our eye: glowing stem cells and new insights into Zika and SCID
Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun. Glowing stem cells help scientists understand how cells work. (Karen Ring) It’s easy to notice when something is going wrong. It’s a lot harder to … Continue reading Stem cell stories that caught our eye: glowing stem cells and new insights into Zika and SCID
Stem cell agency funds clinical trials in three life-threatening conditions
A year ago the CIRM Board unanimously approved a new Strategic Plan for the stem cell agency. In the plan are some rather ambitious goals, including funding ten new clinical trials in 2016. For much of the last year that has looked very ambitious indeed. But today the Board took a big step towards reaching … Continue reading Stem cell agency funds clinical trials in three life-threatening conditions
Advancing Stem Cell Research at the CIRM Bridges Conference
The Bridges program is one of CIRM’s educational initiatives that gives students the chance to take coursework at California state schools and community colleges while conducting stem cell research at top universities and industry labs. The goal is to train the next generation of stem cell scientists by giving them the skills and experience they … Continue reading Advancing Stem Cell Research at the CIRM Bridges Conference
Multi-Talented Stem Cells: The Many Ways to Use Them in the Clinic
CIRM kicked off the 2016 International Society for Stem Cell Research (ISSCR) Conference in San Francisco with a public stem cell event yesterday that brought scientists, patients, patient advocates and members of the general public together to discuss the many ways stem cells are being used in the clinic to develop treatments for patients with … Continue reading Multi-Talented Stem Cells: The Many Ways to Use Them in the Clinic
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
Gene Therapy Beats Half-Matched Stem Cell Transplant in Side-by-Side Comparison to Treat ‘Bubble Baby’ Disease
If you are born with Severe Combined Immunodeficiency (SCID), your childhood is anything but normal. You don’t get to play with other kids, or be held by your parents. You can’t even breathe the same air. And, without treatment, you probably won’t live past your first year. This is the reality of SCID, also called … Continue reading Gene Therapy Beats Half-Matched Stem Cell Transplant in Side-by-Side Comparison to Treat ‘Bubble Baby’ Disease
Potential treatment for sickle cell disease close
Scientists at UCLA found a potentially treatment for sickle cell disease, a new method of gene therapy that can correct the genetic mutation and help the body on its way to generate normal, healthy blood cells for the rest of the patient’s life. The study, funded in part by CIRM and reported in the journal … Continue reading Potential treatment for sickle cell disease close