News

Scientists successfully combined the gene-editing tool CRISPR-Cas9 and a special type of virus to deliver corrected genes to restore the function of the coagulation factor IX (FIX) gene in young mice with hemophilia B. The study, “Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9,”…

Researchers have identified four distinct subgroups of hemophilia A patients according to their profile of factor VIII-targeting antibodies. This may help predict those at risk for developing inhibitors that halt the efficacy of FVIII replacement therapy. The results were presented in a poster titled “Data Coming out of the…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

The relapse rate is higher in older patients with acquired hemophilia A (AHA) treated with immunosuppressive therapies, but it doesn’t affect their overall survival compared with those who don’t relapse, a study suggests. The study, “Relapse pattern and long‐term outcomes in subjects with acquired haemophilia A,” was published in…

Children and adults with hemophilia A in the United States may soon be treated with N8-GP (turoctocog alfa pegol), an engineered formulation of clotting factor VIII developed by Novo Nordisk. The therapy will be available under the brand name Esperoct. The U.S. Food and Drug Administration (FDA) approved N8-GP, or…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders ranging…

SPK-8011 may be a safe treatment strategy for patients with severe or moderately severe hemophilia A, achieving an overall 97% reduction in annual bleeds, according to preliminary data from an ongoing Phase 1/2 trial. The trial (NCT03003533) is intended to explore the safety and efficacy of a single intravenous…

Hemlibra (emicizumab), the sole approved prophylactic treatment for hemophilia A, is now available to patients in developing countries via the World Federation of Hemophilia (WFH) Humanitarian Aid Program after Roche and its subsidiaries Chugai and Genentech joined the program. The donation will provide access to Hemlibra for…

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…

Clinical data from the ongoing Phase 2/3 trial data evaluating MarzAA (marzeptacog alfa activated) in patients with Hemophilia A or B with inhibitors continues to demonstrate the treatment’s potential to prevent bleedings. The most recent trial results were discussed at the 12th Annual Congress of the European Association for Haemophilia…