The U.S. Food and Drug Administration (FDA) has granted fast-track status to SB-525, a clinical gene therapy for hemophilia A developed by Sangamo Therapeutics in partnership with Pfizer. SB-525 delivers a human factor VIII cDNA construct and synthetic liver-specific promoter to the cell’s nucleus by using recombinant adeno-associated virus (rAAV). The…
If you’re active on the Internet, particularly in groups and forums regarding chronic illness, you’ve definitely noticed the term “spoonie” being thrown around. MORE: Although there’s no cure for hemophilia, there are ways that to manage the disease and avoid bleeding episodes. Spoonie is a term coined…
Gentle exercise has many health benefits, particularly for anyone suffering from a chronic illness. In this video, personal trainer Ryan Krane demonstrates some simple exercises that can help counter the effects of chronic fatigue. MORE: Although there’s no cure for hemophilia, there are ways that to manage the disease…
BAY 94-9027, developed by Bayer, is an investigational treatment for severe hemophilia A. It is a PEGylated molecule of recombinant factor VIII in which the B-domain, a region within the factor, is deleted. The therapy is produced using a baby hamster kidney cell line (BHK-21), and has…
BAX 855 (Adynovate) is an approved factor replacement therapy, developed by Baxter International to prevent and treat bleeding in people with hemophilia A. How BAX 855 is manufactured BAX 855 (or rurioctocog alfa pegol) is a PEGylated molecule of full-length recombinant factor VIII (FVIII) produced in Chinese hamster ovary…
The recommended treatment regimen for patients with severe hemophilia to prevent bleeding episodes is to replace the deficient clotting factor. However, currently available replacement clotting factors are limited by their relatively short half-lives and require intravenous injections up to three times a week to maintain protective levels. This can have…
Prophylaxis is commonly referred to as a preventive measure in hemophilia of regularly infusing blood clotting factor concentrates to avoid bleeding. This approach stemmed from research that patients with mild-to-moderate hemophilia (or those who have clotting factor levels of 1 percent or more) rarely experience spontaneous bleeds and have less joint damage…
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…
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