The U.S. Food and Drug administration (FDA) has approved Genentech’s Hemlibra (emicizumab-kxwh) for routine prophylactic (preventive) treatment of patients with hemophilia A without factor VIII inhibitors. Hemlibra is now the sole available prophylactic treatment for patients with hemophilia A with and without factor VIII inhibitors that can…
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights to…
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…
Creative expression within the bleeding disorders community will be on display for the thousands expected to attend the National Hemophilia Foundation’s (NHF) Annual Bleeding Disorders Conference next month in Orlando, Florida. Backed by BioMatrix Specialty Pharmacy, the exhibit, called Blood Work 2018, will showcase interactive gallery submissions from around…
Barry Haarde was a tough man to stop. Despite a decades-long battle with HIV and a near-death brush with hepatitis C, he made history in 2012 as the first person with hemophilia to bike across the country. In fact, he spent the last six years of his life bicycling…
In keeping with its mission to change how members of the bleeding disorder community think about their condition, Octapharma USA has joined with GutMonkey to provide a challenging week-long, water-based experience aimed at boosting patient confidence in managing disease. With an Octapharma grant, 10 participants in “Leading X…
A liver transplant can be live-saving for hemophilia patients, but may not always fully cure the disease, according to the case report about a hemophilia A patient. The letter to the editor describing the report, “Orthotopic liver transplantation for haemophilia A may not…
The investigational gene therapy SB-525 is tolerated well by patients, and dose-dependent responses are positive, according to preliminary results of a Phase 1/2 clinical trial of hemophilia A. The potential gene therapy is being developed as a partnership between Sangamo Therapeutics and Pfizer. It consists of…
About a third of European and North American hemophilia patients are overweight or obese, which is associated with increased joint dysfunction and chronic pain, according to a recent review study. The review, “Obesity in the global haemophilia population: prevalence, implications and expert opinions for weight management,”…
Idelvion (albutrepenonacog alfa), a hemophilia B treatment approved for up to 14-day dosing, is now available in a large, 3500 IU vial size (3500 IU),…
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