Tomorrow, February 28th, is Rare Disease Day. It’s a day to remind ourselves of the millions of people, and their families, struggling with these diseases. These conditions are also called orphan diseases because, in many cases, drug companies were not interested in adopting them to develop treatments. Here at the California Institute for Regenerative Medicine (CIRM), we … Continue reading Join the movement to fight rare diseases
Duchenne muscular dystrophy
High school SPARK intern presents stem cell research to academic audience
Earlier this year, CIRM welcomed many energetic and enthusiastic high school students at the 2022 SPARK Program annual conference in Oakland. The SPARK program is one of the California Institute for Regenerative Medicine’s (CIRM) many programs dedicated to building a diverse and highly-skilled workforce to support the growing regenerative medicine economy right here in California. … Continue reading High school SPARK intern presents stem cell research to academic audience
Stem Cell Agency Board Invests in 19 Discovery Research Programs Targeting Cancers, Heart Disease and Other Disorders
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Dr. Judy Shizuru, Stanford University While stem cell and gene therapy research has advanced dramatically in recent years, there are still many unknowns and many questions remaining about how best to use these approaches in developing therapies. That’s why the governing Board of the California Institute … Continue reading Stem Cell Agency Board Invests in 19 Discovery Research Programs Targeting Cancers, Heart Disease and Other Disorders
HOPE for patients with a muscle destroying disease
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Caleb Sizemore, photo by Todd Dubnicoff Caleb Sizemore says growing up with Duchenne’s Muscular Dystrophy (DMD) was tough. The disease is a rare genetic disorder that slowly destroys a person’s muscles, impairing their ability to walk or breathe. Eventually it attacks the heart leading to premature … Continue reading HOPE for patients with a muscle destroying disease
Joining the movement to fight rare diseases
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST It’s hard to think of something as being rare when it affects up to 30 million Americans and 300 million people worldwide. But the truth is there are more than 6,000 conditions – those affecting 200,000 people or fewer – that are considered rare. Today, … Continue reading Joining the movement to fight rare diseases
A personal reason to develop a better gene therapy
THIS BLOG IS ALSO AVAILABLE AS AN AUDIOCAST ON SPOTIFY Credit : Allison Dougherty, Broad Communications For Sharif Tabebordbar, finding a gene therapy for genetic muscle wasting diseases was personal. When he was a teenager, his father was diagnosed with a rare genetic muscle disease that eventually left him unable to walk. In an interview … Continue reading A personal reason to develop a better gene therapy
Repairing damaged muscles
Close-up of the arm of a 70-year-old male patient with a torn biceps muscle as a result of a bowling injury; Photo courtesy Science Photo Library In the time of coronavirus an awful lot of people are not just working from home they’re also working out at home. That’s a good thing; exercise is a … Continue reading Repairing damaged muscles
Partners in health
From left to right: Heather Dahlenburg, staff research associate; Jan Nolta, director of the Stem Cell Program; Jeannine Logan White, advanced cell therapy project manager; Sheng Yang, graduate student, Bridges Program, Humboldt State University, October 18, 2019. (AJ Cheline/UC Davis) At CIRM we are modest enough to know that we can't do everything by ourselves. … Continue reading Partners in health
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
Two rare diseases, two pieces of good news
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