I am a woman with mild hemophilia. I was angry when I realized that I had gone years without proper diagnosis and treatment. Over the years, I have had multiple bleeds that would have healed more effectively with access to factor replacement products. Many people mourn…

Catalyst Biosciences recently announced that its collaborator, ISU Abixs, has completed dosing of the first subcutaneous cohort in an ongoing Phase 1/2 proof-of-concept clinical trial in people with severe hemophilia B. This patient group is the second of up to five involved in the study. CB 2679d (also known as ISU304)…

The European Commission has approved Hemlibra (emicizumab) for routine preventive treatment of bleeding episodes in people who have hemophilia A with factor VIII inhibitors. The antibody therapy is designed to restore the factor VIII, which is deficient in hemophilia A. Hemlibra is being co-developed by Japan’s Chugai Pharmaceuticals, Switzerland’s…

Acquired hemophilia A (AHA) arose in a 48-year old man with primary biliary cholangitis (PBC), an autoimmune liver disease, according to a case study. While AHA has been associated with autoimmune disorders, the researchers said a connection between AHA and PBC had not been reported previously. While “the…

An experimental form of genome editing called clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a faster and cheaper method of treating people with hemophilia, compared to the current DNA-editing method. Because hemophilia is due to DNA mutations, the genome editing could be used to efficiently slice at the DNA, removing…

Researchers have found that the high price of recombinant clotting factors for patients with hemophilia B is the main cause of the economic burden of the disease in the United States. However, despite the cost, prophylaxis treatment can provide clinical benefits to patients that may reduce other costs not…

All of the hemophilia B patients in an ongoing Phase 1/2 clinical trial of SPK-9001 experienced clotting-factor increases and reductions in bleeding episodes, according to the therapy’s maker, Spark Therapeutics. University of Pennsylvania medical researcher Adam Cuker will present the updated preliminary results today at the Hemostasis and Thrombosis Research Society (HTRS) 2017…

Hurricane Ian, which battered the southeastern United States and the Caribbean in late September and early October, nearly destroyed the home of a client of mine. She compares the damage to a violent tornado combined with a devastating flood. Parts of her home were gutted. In the aftermath, she…

Exercise can be an important part of recovering from hemophilia and continuing to keep a handle on it, according to a German review of previous research. The study, “Programmed Sports Therapy (PST) in People with Haemophilia (PwH) — Sports Therapy Model for Rare Diseases,”  appeared in the…