News

Monthly treatment with the investigational medication fitusiran can reduce bleeding in people with hemophilia A and B, with or without inhibitors, according to data from two Phase 3 clinical trials. “We are encouraged by the data from these initial Phase 3 studies demonstrating fitusiran’s potential as a new…

Physical activities with a high risk of a collision are linked with a greater risk of bleeding in people with moderately severe hemophilia A, a six-month U.S. study reports. This risk was higher for people using on-demand versus preventive replacement factor VIII (FVIII) therapies, the researchers found, and they suggested…

After 18 months, the investigational gene therapy AMT-061 (etranacogene dezaparvovec) continued to prevent bleeds in men with moderate-to-severe hemophilia B, according to top-line data from the Phase 3 HOPE-B clinical trial. The trial has met its pre-specified primary goal, with control of all bleeds by 18 months not…

BioMatrix Specialty Pharmacy has announced this year’s winners of its memorial scholarship program, designed to help people with hemophilia and other bleeding disorders pursue higher education. Six scholarships, each worth $1,000 and honoring people who made “unique” contributions to the bleeding disorder community during their life, are awarded…

Garrett Hayes, who is studying medicine in Texas and has a severe form of hemophilia A, is the first recipient of the National Hemophilia Foundation’s (NHF) Jason Fulton Memorial Scholarship. The scholarship honors the memory of Jason Fulton, a hemophilia patient who died in 1995 at age…

Amarna Therapeutics has secured €5 million (about $5.6 million) in new funding, which it plans to use for developing its lead product, AMA005, an investigational gene therapy that aims to restore blood clotting in people with hemophilia B. The goal is to advance the company’s hemophilia B program…

In the first clinical trial of the experimental therapy SIG-001 for hemophilia A, one patient developed scarring in the spheres used to deliver the therapy that rendered the treatment ineffective, its developer, Sigilon Therapeutics, announced. The Phase 1/2 trial was put on hold by the U.S. Food and Drug…

Preventive treatment with Hemlibra (emicizumab) is safe and can effectively prevent bleeds in people with mild or moderate hemophilia A, according to an interim analysis of data from the HAVEN 6 clinical trial. Results will be presented at the annual meeting of the American Society of Hematology (ASH)…

A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A — and, in 16 of 18 male patients, those levels were sustained for up to four years, according to results from a Phase 1/2 clinical…

A partnership between Emmes and MedGenome seeks to accelerate development of genomics-powered therapies for people with rare diseases, such as hemophilia. The companies plan to combine patient data into custom disease registries to improve research design and help with patient recruitment for clinical trials. In addition, they…