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)…

https://www.youtube.com/watch?v=qKRqJWVQRio This video from the Centers for Disease Control and Prevention takes a look at hemophilia and explains what the blood disorder means for those who suffer from it. MORE: Three tips for coping with a hemophilia diagnosis Hemophilia patients and caregivers share how hemophilia affects their everyday lives…

Researchers have found that adults with severe hemophilia A coupled with pre-existing joint disease benefit most from prophylaxis to improve function, quality of life, and activities, and to better manage pain. The study, “Effect of late prophylaxis in hemophilia on joint status: a randomized trial,” was published in…

The Hemophilia Foundation of American (HFA) has teamed up with the Centers for Disease Control and Prevention (CDC) to create a survey for hemophilia and bleeding disorder patients who don’t currently receive care from a government-funded hemophilia treatment center (HTC). MORE: Why shared data is important for furthering hemophilia research…

Hemophilia is a rare blood disease that usually occurs in males. In fact, it’s extremely rare for women to be born with the condition because of the way it’s passed down genetically. A female would need to inherit two copies of the faulty gene — one from each parent —…

The Boston-based Institute for Clinical and Economic Review (ICER) says a “draft scoping document” outlining its planned comparative review of emicizumab‘s clinical effectiveness and value in treating hemophilia A will be available until Sept. 29. ICER’s report will be subject to a public deliberation in March 2018 by the…

Life with hemophilia can be challenging but there are ways that you can manage the disease to ensure your child leads as normal a life as possible. To help you navigate your child’s illness and help prevent bleeds, we’ve put together a list of tips. MORE: How to prepare for your child’s…

Results of the first group of patients with hemophilia B treated with Catalyst Biosciences’ lead candidate CB 2679d in a Phase 1/2 clinical trial demonstrated its improved stability and activity compared to Pfizer’s BeneFix standard-of-care replacement therapy. CB 2679d (also known as ISU304), was designed to be the…

Hemophilia is a rare blood condition where people do not have the clotting factor that enables their blood to clot when bleeding. It’s an inherited disease that’s usually passed from mother to son. It’s also a disease that’s been prevalent in European royal families. MORE: A brief history of hemophilia…

Dimension Therapeutics may soon become a wholly owned subsidiary of Regenxbio, following the terms of an agreement between the two companies. The merger agreement, if finalized, will add Dimension’s investigational drug candidates being developed to Regenxbio’s pipeline, including DTX201 for the treatment of hemophilia A. DTX201 was being developed…