The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to BioMarin Pharmaceutical’s investigative therapy, valoctocogene roxaparvovec to treat patients with hemophilia A. This new status is likely to speed up the development and regulatory review of valoctocogene roxaparvovec, which has already received an Orphan Drug Designation from both…

An experimental form of genome editing called clustered regularly interspaced short palindromic repeats or CRISPR/Cas9 could offer a faster and cheaper method of treating people with hemophilia, compared to the current DNA-editing method. Because hemophilia is due to DNA mutations, the genome editing could be used to efficiently slice at the DNA, removing…

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to SHP654 (BAX 888), an investigational factor VIII (FVIII) gene therapy candidate being developed by Shire for the treatment of hemophilia A. Gene therapy allows the delivery of a functional copy of the defective gene.

The U.S. Food and Drug Administration (FDA) has approved BioMarin Pharmaceutical‘s Investigational New Drug (IND) application for its gene therapy candidate to treat severe hemophilia A. FDA’s decision on BMN 270, also known for its generic name valoctocogene roxaparvovec,…

Although hemophilia has no cure, there are ways to manage the disease and avoid bleeding episodes. We’ve put together a list of tips to help patients live well with hemophilia using information from the Mayo Clinic and the Centers for Disease Control and Prevention. MORE: World’s…

When you infuse through a port there are several steps to completing an infusion, as opposed to a peripheral stick. Having all the supplies together and prepared and opened in a certain order to maintain good, sterile technique helps make the process move along quickly. Especially when you…

uniQure plans to advance its investigational gene therapy AMT-061 into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B. Gene therapies are being develop to treat diseases caused by genetic mutations. It involves replacing the faulty gene in patients with a healthy gene. AMT-061…

Hemophilia is a rare disorder where the person is unable to stop bleeding due to a lack of certain blood clotting factors. It’s a genetic disease that’s passed down through the X chromosome. Almost all people with hemophilia are male, however, women can be carriers of the disease. MORE: Explaining hemophilia carriers…

Researchers have found that the high price of recombinant clotting factors for patients with hemophilia B is the main cause of the economic burden of the disease in the United States. However, despite the cost, prophylaxis treatment can provide clinical benefits to patients that may reduce other costs not…

In this video from the Hemophilia Federation of America, Kyle McCullough and Jesse Mayhan share what it’s like living with severe hemophilia. The boys discuss their treatment and what they tell friends when they ask about their condition. They also talk about what they’d like to…