Dr. Stephanie Cherqui Last week saw a flurry of really encouraging reports from projects that CIRM has supported. We blogged about two of them last Wednesday, but here’s another two programs showing promising results. UC San Diego researcher Dr. Stephanie Cherqui is running a CIRM-funded clinical trial for cystinosis. This is a condition where patients … Continue reading Two rare diseases, two pieces of good news
Cystinosis Research Foundation
‘A Tornado at the Front Door, a Tsunami at the Back Door’
CIRM funds a lot of research and all of it has life-saving potential. But every once in a while you come across a story about someone benefiting from CIRM-supported research that highlights why the work we do is so important. This story is about a brilliant researcher at UC San Diego developing a treatment for … Continue reading ‘A Tornado at the Front Door, a Tsunami at the Back Door’
“A new awakening”: One patient advocate’s fight for her daughters life
We often talk about the important role that patient advocates play in helping advance research. That was demonstrated in a powerful way last week when the CIRM Board approved almost $12 million to fund a clinical trial targeting a rare childhood disorder called cystinosis. The award, to Stephanie Cherqui and her team at UC San … Continue reading “A new awakening”: One patient advocate’s fight for her daughters life
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
A life-threatening childhood disease and the CIRM-funded team seeking a stem cell cure featured in new video
“My hope for Brooke is she can one day look back and we have to remind her of the disease she once had.” That’s Clay Emerson’s biggest hope for his young daughter Brooke, who has cystinosis, a life-threatening genetic disease that appears by the age of two and over time causes damage to many organs, … Continue reading A life-threatening childhood disease and the CIRM-funded team seeking a stem cell cure featured in new video
The power of the patient’s voice: how advocates shape clinical trials and give hope to those battling deadly diseases
Tennis great Martina Navratilova was once being interviewed about what made her such a great competitor and she said it was all down to commitment. When pressed she said “the difference between involvement and commitment is like ham and eggs; the chicken is involved but the pig is committed.” That’s how I feel about the … Continue reading The power of the patient’s voice: how advocates shape clinical trials and give hope to those battling deadly diseases
The Stem Cellar 