A single administration of AMT-061, an investigational gene therapy for people with severe and moderately severe hemophilia B, increased therapeutic levels of factor IX (FIX) in all patients enrolled in uniQure’s ongoing Phase 2b trial, the company announced. Ultimately, this trial’s purpose is to confirm the dose of AMT-061…
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.
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…
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…
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history — how disorders such as spinal muscle atrophy (SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials,…
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…
Clinical symptoms of acquired hemophilia A in elderly patients can vary widely, a series of six case reports reveals. Also, these patients should be managed individually in order to achieve the best care and outcome, researchers suggest. The cases were described in the study “Management of…
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…
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…
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,”…
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