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UrduAdults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published today in the New England Journal of Medicine by researchers from the University of Pennsylvania Perelman School of Medicine and a multicenter group of investigators.
Tag: Hemophilia
ISTH Releases Evidence-Based Clinical Practice Guideline for Hemophilia Treatment
The International Society on Thrombosis and Haemostasis (ISTH), the leading international professional medical-scientific organization dedicated to advancing the understanding, prevention, diagnosis, and treatment of conditions related to thrombosis and hemostasis, has published the first clinical practice guideline utilizing strict GRADE methodology for the treatment of congenital hemophilia A and B.
McMaster University researchers trial potential hemophilia treatment
The trial, which concluded in 2021, found that 133 hemophilia patients treated weekly with injections of efanesoctocog alfafor a year improved their outcomes and quality of life compared to their previous treatment.
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.
Children’s Hospital of Philadelphia Awards Gold Medal to Dr. Katherine High, Gene Therapy Pioneer
In an honor reserved for only the most significant achievements in advancing children’s health, Children’s Hospital of Philadelphia (CHOP) has awarded its Gold Medal to Katherine High, MD, a gene therapy pioneer and one of the lead developers of the first in vivo gene therapy approved by the Food and Drug Administration (FDA). Bestowed only 12 times in CHOP’s 167-year history, the Gold Medal highlights Dr. High’s groundbreaking discoveries at CHOP, which led to a gene therapy treatment for a rare form of inherited blindness and advanced gene therapy for hemophilia to late-stage testing.
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.