News

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies(NCATS) at the…

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…

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…

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…

A gene therapy being developed by ASC Therapeutics restored levels of the clotting factor missing in hemophilia A in a mouse model of the disease, and further tests in primates and human cells suggest the therapy might also work for people, the company announced. Results from…

Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.

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…

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…