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
RP-L201
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
Promising results from CIRM-funded projects
Severe Leukocyte Adhesion Deficiency-1 (LAD-1) is a rare condition that causes the immune system to malfunction and reduces its ability to fight off viruses and bacteria. Over time the repeated infections can take a heavy toll on the body and dramatically shorten a person’s life. But now a therapy, developed by Rocket Pharmaceuticals, is showing … Continue reading Promising results from CIRM-funded projects