FLT180a for hemophilia
FLT180a, also known as verbrinacogene setparvovec, is an investigational gene therapy that was investigated as a treatment for people with hemophilia B.
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Higher Doses of Adynovate is Effective as Preventive Treatment for Hemophilia A, Trial Suggests
Maintaining high concentrations of Adynovate in the blood of hemophilia A patients decreases the number of bleedings and enables more patients to stop having bleeds, according to the first results of a Phase 3b/4 study by Takeda.
My Son Progressed from Severe Hemophilia to Mild, and I’ll Take It
My mighty warrior Caeleb is about to start a new chapter in his life with hemophilia; it’s one I never imagined I’d see. About nine months ago, Caeleb began a new treatment with a medication that has been taking the bleeding disorder world by storm. Hemlibra (emicizumab-kxwh)…
First Patient Treated in Phase 3 Trial of AMT-061 in Hemophilia B
A Phase 3 clinical trial of treatment candidate AMT-061 for severe and moderately severe hemophilia B has treated its first patient. The open-label HOPE-B study (NCT03569891) is testing the safety and effectiveness of AMT-061 in adult men with hemophilia B. Patient recruitment is continuing for an estimated total of 56…
Research into Physiotherapy and Rehab for Hemophilia Patients Needed, Review Says
People with hemophilia may benefit from physiotherapy and rehabilitation programs intended to manage bleeding and joint disease (arthropathy), but more research is needed to better understand those programs — and their duration — that might be of most help, a review study says. The review, “Recent advances in musculoskeletal…
Study Finds No Differences in Oral Health Quality of Life in Children With Hemophilia
Despite having lower oral health scores, children with hemophilia showed no differences in oral health-related quality of life compared to children without the condition, a study found. The study, “Parent’s report on oral health‐related quality of life of children with haemophilia,” was published in Haemophilia. Hemophilia is a…
There Is Lifesaving Wisdom and Care in the Hemophilia Community
Thank goodness for the great friendships that we forge along the way. Those of us who live with chronic illnesses learn the most about caring for our loved ones from those who are in our communities. While teaming with medical staff is crucial to the diagnosis and treatment…
Cell-based Gene Therapy May Offer Long-term Treatment for Hemophilia A, Mouse Study Suggests
Cell therapy with endothelial progenitor cells and stem cells genetically engineered to produce a functional clotting factor VIII (FVIII) may provide a stable and long-term treatment for hemophilia A, a mouse study has found. The study shows that the transplant of cells…
Low Doses of Feiba Cut Risk of Relapse After Acute Bleeding Events in Acquired Hemophilia A, Study Says
Administering low doses of Feiba, or activated prothrombin complex concentrate (aPCC), as a prophylactic (preventive) treatment after acute bleeding events reduces the risk of relapse in patients with acquired hemophilia A, a study shows. The study “Low dose of IPCC after the initial treatment in acquired…
ISTH-BAT Is Useful to Identify, Assess Hemophilia Patients, Study Suggests
The International Society on Thrombosis and Hemostasis (ISTH) created a Bleeding Assessment Tool (BAT) that is useful to identify and assess disease severity in people with hemophilia, both newly diagnosed patients and those who had a prior diagnosis, a study found. The study, “Application of the ISTH bleeding score in hemophilia,”…