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…
This month, we celebrated the two holidays of Easter and Passover. Many of my Christian friends describe Easter as a celebration of freedom over sin and death due to the resurrection of Jesus Christ. My Jewish friends gather to recount the story of Passover, a celebration of freedom from slavery…
A gene therapy-based factor VIIa (FVIIa) given as prophylactic, or preventative, treatment was seen to produce specific factor levels needed to reduce the frequency of spontaneous bleeding episodes in a rat model of hemophilia A. According to scientists, this work may be a first step in developing therapies that…
Get regular updates to your inbox.
