Adults 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...
University of PennsylvaniaSep 25 2024 Adults 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.
"What we saw from patients in this study was that within a few days of receiving the gene therapy infusion, it took root, and their bodies started making factor IX for the first time in their lives," said study investigator and lead author Adam Cuker, MD, MS, section chief for Hematology, and clinical director of the Penn Blood Disorders Center and the Penn Comprehensive Hemophilia Program.
FDA-approved gene therapies available at Penn Medicine Based on the results of this study, the FDA approved the gene therapy in April 2024. Cuker was the site lead for the clinical trial at Penn Medicine, which was one of the top-enrolling sites for the study. It represents the second form of gene therapy approved to treat hemophilia B.
Related StoriesIn the current study, the most common adverse effect was related to an immune system attack on liver cells that were targeted by the gene therapy, which can render the gene therapy ineffective, if not quickly treated. In the study, affected patients were treated with steroids to limit this immune reaction. Patients in the study will continue to be followed for at least five years to monitor potential long-term side effects.
Gene Therapy Hemophilia Hemophilia B Bleeding Blood Clinical Trial Genetic Genetic Disorder Liver Medicine
United Kingdom Latest News, United Kingdom Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
Mobile-based tool to address heavy drinking in young adults passes pilot phaseYoung adults who drink heavily reported that a specific intervention that provides personalized feedback on daily drinking would be beneficial for people who want to reduce their drinking and would encourage self-reflection among those who are not yet ready to change their drinking habits.
Read more »
COVID-19 linked to increased risk of hearing loss in young adults, study findsResearchers in South Korea found that young adults who recovered from COVID-19 are at significantly higher risk of experiencing hearing loss and sudden sensorineural hearing loss compared to those who never had the virus.
Read more »
Study reveals harrowing experiences of misdiagnosis of borderline personality disorder in autistic adultsConducted through interpretive phenomenological analysis, a study published in the journal Autism involved one-to-one, semi-structured interviews with 10 participants. Each participant recounted unnoticed autistic traits from childhood and described the pervasive impact of a BPD misdiagnosis.
Read more »
Study highlights limited evidence for antidepressants in treating pain in older adults Univer...University of Sydney research has found people over 65 are being prescribed antidepressants as pain treatment based on international guidelines that use limited evidence.
Read more »
Hull teenager who beat brain tumour to study medicineAged 10, Harrison Sherwell underwent treatment after doctors found a cancerous tumour.
Read more »
RSV vaccination in older adults with health conditions is cost-effective, modeling study findsTargeting vaccination programs for respiratory syncytial virus (RSV) to older adults with underlying health conditions is a cost-effective way to reduce disease, according to a new modeling study in the Canadian Medical Association Journal (CMAJ).
Read more »