Signs are appearing outside restrooms in the United Kingdom to advise members of the public that not all disabilities are visible. MORE: Learn more about hemophilia and what it really means to have the blood disorder. According to a report from the BBC, the signs are being posted to help those…

The National Center on Birth Defects and Development Disabilities has an online booklet that helps hemophiliacs and their families construct a plan in the event of an emergency. MORE: Initial results from phase 3 hemophilia trial of Emicizumab in children looks promising. The plan focuses on all aspects…

Shire has launched a Phase 3 clinical trial (NCT02585960) with Adynovate, a drug approved to help treat and control bleeding in children and adults with hemophilia A. The trial is currently recruiting participants. Adynovate is an injectable recombinant factor VIII (FVIII), designed using pegylation technology, which allows it to last…

A Phase 3 extension trial has confirmed Alprolix’s long-term ability to prevent bleeding episodes in hemophilia B patients, and its safety. Those in the B-YOND extension trial did not develop an immune response against Alprolix. Adults and adolescents were treated for three years, and children under 12 for 18 months. “These results…

A hemophilia patient has died in a Phase 3 trial evaluating Roche’s  experimental treatment emicizumab (ACE910), raising further concern about its safety. Preliminary indications are that the patient died of a rectal hemorrhage and not emicizumab therapy, however, the company said. The death follows reports of serious blood-clotting events in…

An analysis was highly critical of the Orphan Drug Act, used by the U.S. Food and Drug Administration since 1983 to stimulate research into rare diseases. The study found that the act is not “sufficiently” effective in meeting the needs of patients with less common diseases, like hemophilia, while pharmaceutical companies are finding it profitable,…

Sangamo Therapeutics recently presented what it characterized as key improvements to its technology platform for engineering highly specific zinc finger nucleases (ZFNs), which are used for targeted editing of the human genome. This technique has broad applicability, namely for the treatment of hemophilia B and other disorders. The…

The U.S. Food and Drug Administration (FDA) has approved Shire’s Adynovate for the treatment of pediatric patients under 12 years of age with hemophilia A. Adynovate also was approved for use by both adults and children undergoing surgical procedures, to control bleeding episodes. Adynovate is a recombinant Factor…

Biogen recently hired two more executive management team members for Bioverativ, a Biogen spin-off company that is expected to be up and running in early 2017. Bioverativ will focus on the discovery, research, development, and commercialization of new treatments for hemophilia and other blood disorders. Rogerio Vivaldi, MD, is Bioverativ’s new executive…

NuFACTOR is accepting applications for the 2017 Annual Eric Dostie Memorial College Scholarship. NuFACTOR is a specialty pharmacy of FFF Enterprises, a distributor of plasma products, vaccines and biosimilars, among others. NuFACTOR specializes in the treatment of chronic conditions in several areas of medicine, including blood disorders and…