Brenden Whittaker Brenden Whittaker was born with a rare genetic disorder called X-linked chronic granulomatous disease (X-CGD). This condition affects the immune system's ability to fight off common germs, specifically bacteria and fungi, and can result in infections that would only be mild for healthy people. Unfortunately for Brenden, he has suffered life-threatening infections that … Continue reading First patient in CIRM funded X-CGD trial gives back by working in patient care
Rare Diseases
Exploring tough questions, looking for answers
COVID-19 and social and racial injustice are two of the biggest challenges facing the US right now. This Thursday, October 8th, we are holding a conversation that explores finding answers to both. The CIRM Alpha Stem Cell Clinic Network Symposium is going to feature presentations about advances in stem cell and regenerative research, highlighting treatments … Continue reading Exploring tough questions, looking for answers
CIRM Bridges program prepared student for research of a rare disease
Ian Blong, Ph.D., CIRM San Francisco State University Bridges to Stem Cell Research Alumnus Recently, The New York Times released a powerful article that tells the stories of four different families navigating the challenges of having a family member with a rare disease. One of these stories focused on Matt Wilsey, a tech entrepreneur and … Continue reading CIRM Bridges program prepared student for research of a rare disease
It’s all about the patients
Ronnie, born with a fatal immune disorder now leading a normal life thanks to a CIRM-funded stem cell/gene therapy: Photo courtesy of his mum Upasana Whenever you are designing something new you always have to keep in mind who the end user is. You can make something that works perfectly fine for you, but if … Continue reading It’s all about the patients
CIRM-funded kidney transplant procedure eyeing faster approval
Kidney transplant surgery. Medeor Therapeutics, which is running a CIRM-funded clinical trial to help people getting kidney transplants, just got some really good news. The US Food and Drug Administration (FDA) has just granted their product Regenerative Medicine Advanced Therapy (RMAT) designation. That’s a big deal because it means they may be able to apply … Continue reading CIRM-funded kidney transplant procedure eyeing faster approval
Building a progressive pipeline
Dr. Kelly Shepard By Dr. Kelly Shepard One of our favorite things to do at CIRM is deliver exciting news about CIRM projects. This usually entails discussion of recent discoveries that made headlines, or announcing the launch of a new CIRM-funded clinical trial …. tangible signs of progress towards addressing unmet medical needs through advances … Continue reading Building a progressive pipeline
Meet the people who are changing the future
Kristin MacDonald Every so often you hear a story and your first reaction is “oh, I have to share this with someone, anyone, everyone.” That’s what happened to me the other day. I was talking with Kristin MacDonald, an amazing woman, a fierce patient advocate and someone who took part in a CIRM-funded clinical trial … Continue reading Meet the people who are changing the future
A new voice and vision added to CIRM Board
UC Davis School of Medicine Dean, Dr. Allison Brashear: Photo courtesy UCD We have a new member on the CIRM Board – Dr. Allison Brashear is the Dean of the UC Davis School of Medicine, overseeing one of the nation’s top research, academic and medical training institutions. Dr. Brashear is an internationally known researcher in … Continue reading A new voice and vision added to CIRM Board
Cord blood transplants help children fighting deadly diseases
Dr. Paul Szabolcs: Photo courtesy of UPMC A simple blood stem cell transplant is showing tremendous promise in treating a wide range of metabolic, blood and immune disorders such as thalassemia and some leukodystrophies. These are considered rare diseases – meaning there are fewer than 200,000 people with them in the US – so there … Continue reading Cord blood transplants help children fighting deadly diseases
A Tribute to Huntington’s Disease Warriors in the Age of COVID-19
Frances Saldana is one of the most remarkable women I know. She has lost all three of her children to Huntington's disease (HD) - a nasty, fatal disease that steadily destroys the nerve cells in the brain - but still retains a fighting spirit and a commitment to finding a cure for HD. She is … Continue reading A Tribute to Huntington’s Disease Warriors in the Age of COVID-19