News

After handing over the development of the gene therapy candidate SB-525 to Pfizer, Sangamo Therapeutics has now concluded its collaboration agreement with Biogen to advance and market gene therapies for Alzheimer’s and Parkinson’s diseases, and other neuromuscular and other neurological disorders. Among other goals, the…

In contrast to observations in the general population, blood type and levels of von Willebrand factor (VWF) don’t appear to influence factor VIII (FVIII) levels in people with non-severe hemophilia A, a study reports. Moreover, age was found to play only a minor role in FVIII levels in…

Due to the global COVID-19 pandemic, World Hemophilia Day (WHD) will be  a little different this year to keep everyone safe. Still, there are multiple ways to participate in the April 17 event intended to increase awareness and understanding of hemophilia and other bleeding disorders. Begun in 1989 and…

After receiving guidance from both the U.S. Food and Drug Administration and the European Medicines Agency, Catalyst Biosciences is preparing to launch a pivotal Phase 3 clinical trial assessing marzeptacog alfa activated (MarzAA) for the treatment of bleeding episodes in people hemophilia A or B with…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

More opioids were prescribed to treat pain in adults and children with hemophilia than was reported in U.S. national datasets, a new study shows. As such use carries a risk for opioid‐related complications, hemophilia treatment centers (HTC) need to take a more active role in pain management…

Novo Nordisk reports that its treatments for hemophilia and other disorders remain available, and it is taking measures to support patients during the current COVID-19 pandemic. The company has large stock of medicines and expects to be able to meet pharmacy requests in the U.S. during the…

The U.S. Food and Drug Administration (FDA) approved Sevenfact (coagulation factor VIIa [recombinant]-jncw) for the treatment and control of spontaneous bleeding episodes in people, ages 12 and older, with hemophilia A or B with inhibitors. Hemophilia, a genetic disorder, affects the body’s ability to make blood…

Although obese people with hemophilia — as well as their spouses and caregivers — are aware of the risks of excessive weight, fewer than half are actively taking steps to lose weight, a new study shows. Better support and education about weight management would be beneficial for those patients, the…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…