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
Rare Disease Day
How developing a treatment for a rare disease could lead to therapies for other, not-so-rare conditions
Logan Lacy, a child with AADC Deficiency: Photo courtesy Chambersburg Public Opinion Tomorrow, the last day in February, is Rare Disease Day. It’s a day dedicated to raising awareness about rare diseases and the impact they have on patients and their families. But the truth is rare diseases are not so rare. There are around … Continue reading How developing a treatment for a rare disease could lead to therapies for other, not-so-rare conditions
Rare Disease Day – fighting for awareness and hope
It’s hard thinking of something as rare when one in 20 people are at risk of experiencing it in their lifetime. But that’s the situation with rare diseases. There are more than 7,000 of them and each affects under 200,000 people. In some cases they may only affect a few hundred people. But for each … Continue reading Rare Disease Day – fighting for awareness and hope
Breaking the isolation of rare diseases
How can something that affects 30 million Americans, one in ten people in the US, be called rare? But that’s the case with people who have a rare disease. There are around 7,000 different diseases that are categorized as rare because they affect fewer than 200,000 people. Less than five percent of these diseases have … Continue reading Breaking the isolation of rare diseases
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
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
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
Rare Disease Day, a chance to raise awareness and hope.
Battling a deadly disease like cancer or Alzheimer’s is difficult; but battling a rare and deadly disease is doubly so. At least with common diseases there is a lot of research seeking to develop new treatments. With rare diseases there is often very little research, and so there are fewer options for treatment. Even just … Continue reading Rare Disease Day, a chance to raise awareness and hope.
The Stem Cellar 