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UrduChildren’s Hospital Los Angeles (CHLA), a national leader in advanced pediatric medical care and innovative cell and gene therapy research and treatment, announced today the addition of its 10th cell and gene therapy treatment, establishing CHLA as the pediatric provider offering the most FDA-approved, state-of-the-art cell and gene therapy treatments on the West Coast.
Tag: Hemophilia A
Tulane expert lauds new ‘cutting edge’ hemophilia treatment
A new treatment that helps people with hemophilia A maintain higher levels of a crucial blood clotting factor with fewer treatments is a victory for patients, according to a new editorial in The New England Journal of Medicine this week by Dr. Cindy…
Liver cancer study encourages caution with certain gene therapies
A newly discovered link between protein misfolding and liver cancer could help improve gene therapy for hemophilia.
CHOP-led Research Study Identifies Key Target in Treatment-Resistant Hemophilia A
Researchers at Children’s Hospital of Philadelphia (CHOP) have identified a key target that may be responsible for treatment failure in about 30% of patients with hemophilia A. The target, known as B cell activating factor (BAFF), appears to promote antibodies against and inhibitors of the missing blood clotting factor that is given to these patients to control their bleeding episodes. The findings, published in the Journal of Clinical Investigation, raise the possibility of using anti-BAFF therapies, potentially in combination with immune tolerance therapies, to tame the immune response in some patients with severe hemophilia A.