U.S. doctors caring for people with hemophilia are more likely to make treatment decisions based on patient preferences than doctors in the U.K., who tend to be more influenced by their colleagues and government policies, according to a study comparing decision-making in both…
Sigilon Therapeutics’ candidate cell therapy for hemophilia A, called SIG-001, delivers sustained production of factor VIII for over six months and corrects bleeding in a hemophilia A mouse model. These results were shared at the 2019 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, in a…
A single infusion of AMT-061, an investigational gene therapy for hemophilia B, continues to maintain clinically significant increases in clotting factor IX (FIX) up to six months after its administration, as demonstrated in all three patients enrolled in uniQure…
I don’t think anyone likes having a chronic disease. I have good days, and I have bad days. Thankfully, the good days outnumber the bad ones. There are days when I tolerate having hemophilia, days when I can step back and see the gifts that have come from…
Trio Health has launched a novel database with real-time clinical data of patients with hemophilia which combines information from medical doctors and pharmacies. The main aim of the database is to provide information on the treatment regimen to physicians in order to improve patients’ outcomes. Access to the database…
A new collaboration between St. Jude Children’s Research Hospital and the World Federation of Hemophilia (WFH) is aimed at helping blood disorder patients in countries where they may not receive regular treatment. The partnership’s centerpiece will be a program to conduct a gene therapy clinical trial in…
Hemophilia B Leyden (HBL) is a sub-type of hemophilia B, a genetic bleeding disorder characterized by a lack of a blood-clotting protein called factor IX. HBL accounts for approximately 3% of all hemophilia B cases. HBL is distinct from other forms of hemophilia because, while it is caused by…
Hemophiliacs in Europe have never had so many reasons to feel hopeful about the future, thanks to significant recent scientific advances. But “vested interests, entrenched systems, and blatant, outright corruption limit access” to even basic healthcare in some Eastern European countries, says Amanda Bok, CEO of the Brussels-based European…
A single dose of the experimental gene therapy AMT-180 promotes clinically meaningful blood-clotting activity, independent of factor VIII levels, in mouse and primate models of hemophilia A, a study shows. These preclinical findings were discussed in a presentation, “Towards AAV5-Mediated Gene Therapy for Hemophilia A with a Factor IX…
Point-of-care ultrasonography enables improved diagnostic accuracy, and allows for more targeted treatment and better monitoring of therapy response in hemophilia patients with joint complications, a study suggests. The study, a review of published articles, “Point-of-care Ultrasonography in Orthopedic Management of Hemophilia: Multiple Uses of an Effective Tool,” was published…
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