News

Diabetes and infection with human immunodeficiency virus (HIV) increase the risk of high blood pressure (hypertension) among men with hemophilia, a U.S. study found. Given that both hypertension and diabetes can lead to a brain bleed in hemophilia patients, these findings support early screening for these conditions in this…

Hepatitis C virus (HCV) infection remains a major risk factor for hepatocellular carcinoma — the most common type of liver cancer — in men with hemophilia, despite the wide availability of medications to fight the virus, a U.S. database analysis has found. Other independent risk factors for the cancer…

A telerehabilitation program, wherein physical therapy exercises were delivered to patients by a professional via video calls, was deemed safe and effective at improving the physical condition of people with severe hemophilia, according to a small study in Chile. Patients reported a high degree of satisfaction with the intervention,…

A single dose of Freeline Therapeutics’ experimental gene therapy FLT180a increased the levels of factor IX (FIX) — the missing clotting protein in hemophilia B — for up to around 3.5 years in nine out of 10 men with moderate-to-severe disease. That’s according to data from the Phase…

Most people with hemophilia A or B who switched from an approved prophylactic (preventive) therapy regimen to once-monthly treatment with fitusiran in the ATLAS-PPX clinical trial had no bleeds over seven months on the investigational therapy, new data show. Fitusiran also reduced bleeding rates regardless of the presence…

Weekly use of the experimental therapy efanesoctocog alfa maintains therapeutic levels of factor VIII (FVIII) — the missing clotting factor in hemophilia A — and significantly improves physical and joint health, while reducing pain, in adolescents and adults with a severe form of the disease. These are the…

The first three participants in the B-LIEVE trial all have factor IX (FIX) levels within the normal range more than one month after being treated with FLT180a, Freeline’s experimental gene therapy for hemophilia B, according to the company. “The initial data show that FLT180a provides rapid and consistent…

Once-daily preventive treatment with Novo Nordisk’s experimental therapy concizumab significantly reduced bleeding rates in people with both hemophilia A and B with inhibitors, according to new data from the Phase 3 explorer7 trial. In fact, bleeding rates in patients receiving concizumab in trial decreased by more than 85%. Based…

Mim8, an investigational therapy to prevent bleeding episodes in people with hemophilia A, is safe and well-tolerated at multiple doses, according to data from the Phase 1/2 FRONTIER1 trial. The therapy also showed signs of efficacy, with most patients given higher doses experiencing no bleeds during treatment. “We are…

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…