You can read about a disease or hear someone talking about it and be engaged and interested. But when you see and hear the people who have the disease talking about it and the impact it has on their lives, that's when a profound impact occurs. When you look into their eyes and hear them … Continue reading Using film to break down barriers around rare disease
rare disease
Rare disease gets go-ahead to run clinical trial
Cystinosis is one of those diseases most people have never heard of and should be very grateful they haven't. It's rare - affecting only around 500 children and young adults in the US and just 2,000 people worldwide - but it's nasty. Up to now the treatments for it have been very limited. But a … Continue reading Rare disease gets go-ahead to run clinical trial
Stem Cell Agency’s supporting role in advancing research for rare diseases
The recent agreement transferring GSK’s rare disease gene therapies to Orchard Therapeutics was good news for both companies and for the patients who are hoping this research could lead to new treatments, even cures, for some rare diseases. It was also good news for CIRM, which played a key role in helping Orchard grow to … Continue reading Stem Cell Agency’s supporting role in advancing research for rare diseases
Bridging the divide: stem cell students helping families with rare diseases become partners in research
Sometimes it’s the simplest things that make the biggest impact. For example, introducing a scientist to a patient can help them drive stem cell research forward faster than either one could do on their own. Want proof? This year, students in CIRM’s Bridges to Stem Cell Research and Therapy program at California State University (CSU) … Continue reading Bridging the divide: stem cell students helping families with rare diseases become partners in research
Raising awareness about Rare Disease Day
One of the goals we set ourselves at CIRM in our 2016 Strategic Plan was to fund 50 new clinical trials over the next five years, including ten rare or orphan diseases. Since then we have funded 13 new clinical trials including four targeting rare diseases (retinitis pigmentosa, severe combined immunodeficiency, ALS or Lou Gehrig’s … Continue reading Raising awareness about Rare Disease Day
Partnering with the best to help find cures for rare diseases
As a state agency we focus most of our efforts and nearly all our money on California. That’s what we were set up to do. But that doesn’t mean we don’t also look outside the borders of California to try and find the best research, and the most promising therapies, to help people in need. … Continue reading Partnering with the best to help find cures for rare diseases
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 Cells Profile in Courage: Pat Furlong, Patient Advocate
One of the true joys for me in helping put together this year’s Annual Report was getting to know the patients and patient advocates that we profiled in the report. These are some extraordinary individuals and the short profiles we posted only touch the surface of just how extraordinary. So, over the next few weeks … Continue reading Stem Cells Profile in Courage: Pat Furlong, Patient Advocate
Meeting the scientists who are turning their daughter’s cells into a research tool – one that could change her life forever
There’s nothing like a face-to-face meeting to really get to know someone. And when the life of someone you love is in the hands of that person, then it’s a meeting that comes packed with emotion and importance. Last week Gay and Steve Grossman got to meet the people who are working with their daughter … Continue reading Meeting the scientists who are turning their daughter’s cells into a research tool – one that could change her life forever
How research on a rare disease turned into a faster way to make stem cells
If Forest Gump were a scientist, I’d like to think he would have said his iconic line a little differently. Dr. Gump would have said, “scientific research is like a box of chocolates - you never know what you’re gonna get.” A new CIRM-funded study coming out of the Gladstone Institutes certainly proves this point. … Continue reading How research on a rare disease turned into a faster way to make stem cells
The Stem Cellar 