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…
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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,”…
Repetitive movements, fitness level, and prolonged and competitive physical activity are among the factors that raise bleeding risks for people with hemophilia taking part in such activities, a study based on a survey of physical therapists in the U.S. reports. Duration of training and safety equipment were seen as modifiable factors,…
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Sigilon, MIT Collaborating to Develop Programmable Cell Therapies for Hemophilia, Other Diseases
Sigilon Therapeutics and the Massachusetts Institute of Technology will work together to develop programmable cell therapeutics for the treatment of hemophilia, lysosomal storage diseases, and other serious chronic illnesses. The collaboration will combine Sigilon’s proprietary cell technology with synthetic biology approaches developed by Ron Weiss, PhD, a…
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