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
Leukocyte Adhesion Deficiency-I
Positive results for patients enrolled in CIRM-funded trial of a rare pediatric disease
Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease that prevents patients from combating infections. This leads to recurring bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations, and can be fatal. It is caused by a mutation in a specific gene that causes low levels of a protein called CD18. The … Continue reading Positive results for patients enrolled in CIRM-funded trial of a rare pediatric disease
Going the extra mile to save a patient’s life
You can tell an awful lot about a company by the people it hires and the ability it gives them to do their job in an ethical, principled way. By that measure Rocket Pharma is a pretty darn cool company. Rocket Pharma is running a CIRM-funded clinical trial for Leukocyte Adhesion Deficiency-I (LAD-I), a rare … Continue reading Going the extra mile to save a patient’s life
Positive results from CIRM-funded LAD-I trial presented at the 62nd American Society of Hematology Annual Meeting
Gaurav Shah, M.D., CEO and President of Rocket Pharmaceuticals Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease caused by a mutation in a specific gene that causes low levels of a protein called CD18. Due to low levels of CD18, the adhesion of immune cells is affected, which negatively impacts the body’s ability to … Continue reading Positive results from CIRM-funded LAD-I trial presented at the 62nd American Society of Hematology Annual Meeting
CIRM funded trial for LAD-I announces positive results
Leukocyte Adhesion Deficiency-I (LAD-I) is a rare pediatric disease caused by a mutation in a specific gene that causes low levels of a protein called CD18. Due to low levels of CD18, the adhesion of immune cells is affected, which negatively impacts the body’s ability to combat infections. Rocket Pharmaceuticals has announced positive results from … Continue reading CIRM funded trial for LAD-I announces positive results
Four CIRM Funded Trials Release Results at 2019 ASH Meeting
With more than 17,000 members from nearly 100 countries, the American Society of Hematology (ASH) is an organization composed of clinicians and scientists around the world working to conquer various blood diseases. Currently, they are having their 61st Annual ASH Meeting to highlight some of the exciting work going on in the field. Four of … Continue reading Four CIRM Funded Trials Release Results at 2019 ASH Meeting
CIRM Board Approves Funding for New Clinical Trials in Solid Tumors and Pediatric Disease
Dr. Theodore Nowicki, physician in the division of pediatric hematology/oncology at UCLA. Photo courtesy of Milo Mitchell/UCLA Jonsson Comprehensive Cancer Center The governing Board of the California Institute for Regenerative Medicine (CIRM) awarded two grants totaling $11.15 million to carry out two new clinical trials. These latest additions bring the total number of CIRM funded … Continue reading CIRM Board Approves Funding for New Clinical Trials in Solid Tumors and Pediatric Disease
The Stem Cellar 