A single dose of the experimental gene therapy FLT180a can safely maintain the activity levels of factor IX (FIX) within a normal range for nearly three years, effectively preventing bleeds and the need for replacement therapies in patients with hemophilia B, according to updated data from a Phase 1/2…

Dosing has been resumed in ongoing clinical trials in the U.S. testing the investigational medication fitusiran in adults and adolescents with hemophilia, Sanofi announced in a press release. The company had placed a voluntary dosing hold on its full clinical development program for fitusiran at the end of…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to marzeptacog alfa activated (MarzAA), a lab-made version of clotting factor VIIa for the subcutaneous (under-the-skin) treatment of episodic bleeds in hemophilia A and B patients with inhibitors. MarzAA, developed by Catalyst Biosciences, will…

AMT-060, a first-generation gene therapy candidate by uniQure, continues to safely reduce the number of bleeding episodes and the use of clotting factor IX (FIX) in men with moderate to severe hemophilia B, long-term data from a Phase 1/2 clinical trial show. The therapy also prevented…

Most people with hemophilia have a positive attitude toward gene therapy, and are willing or “very willing” to receive this treatment, according to a small interview study assessing patient perceptions. These patients reported that they choose their treatments based mainly on five criteria. Specifically, those are…

I visited a hemophilia treatment center for the first time in the 1980s, to have my factor levels tested. The phlebotomist couldn’t find a vein, and I was distraught. Workers ended up pinning me down to get my blood. The result was that my factor…

A Phase 4 trial investigating Ixinity as a prophylactic treatment for children under age 12 with hemophilia B has reached the halfway mark for its enrollment target, Medexus Pharmaceuticals announced. The international study (NCT03855280), taking place at sites in Brazil, Colombia, Moldova, South Africa, and Ukraine, continues recruiting eligible…

The Dietary Approach to Stop Hypertension (DASH) diet yields beneficial health effects, reducing blood pressure, sugar, and fat, and increasing high-density lipoprotein (HDL) levels or so-called “good” cholesterol, in adolescents with hemophilia, an Iranian…

Manual therapy, in combination with passive stretching, can reduce the frequency of joint bleeds and improve joint health in people with hemophilia, a small study shows. The study, “The effectiveness of manual therapy in addition to passive stretching exercises in the treatment of patients with haemophilic…

Using a combination of genetic and functional approaches, scientists have identified new mutations in parts of the F8 gene that do not code for factor VIII (FVIII) in a small group of French and Canadian patients with hemophilia A. These results were described in the study, “The…