Researchers at South Dakota State University (SDSU) studying mouse sperm found that drugs such as those used to treat HIV infection suppress L1s, or snippets of DNA that cause gene mutations. L1s are called transposons or mobile genetic elements. They are DNA fragments in the genome that replicate themselves and…

The teenage years are tough for most children but for those living with a chronic bleeding disorder, these years can be extra stressful. When they’re young, most of a hemophilia patient’s needs are met by their parents and they can be suitably supervised to ensure they come to no harm. However,…

Some hemophilia patients may, over time, develop inhibitors which restrict the efficacy of treatment making bleeding episodes more difficult to stop. MORE: Intracranial hemorrhage risk – when to see a doctor According to the Centers for Disease Control and Prevention (CDC), although the majority of people living with…

When young Max Levy had to have a port put in his chest for hemophilia medication, his family nicknamed him Iron Man after one of his favorite Marvel action heroes. The story touched a lot of hearts and was even shared by Robert Downey Jr. on his…

BioMarin Pharmaceutical  will add an additional Phase 3 study to the clinical development of its investigational gene therapy BMN 270 for hemophilia A. BioMarin will conduct two separate trials testing the effectiveness and safety of two different doses of BMN 270. Both trials are expected to initiate in the…

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…

A new hemophilia management program was launched as part of a collaborative agreement between Magellan Rx Management and Health New England to standardize treatment practices and optimize pharmacy and medical benefits use and costs. The goal is to provide better overall quality of care at reduced costs. Among…

There are organizations all over the world that are dedicated to helping people with hemophilia and their loved ones. To make your search a little easier, we’ve listed some of the the more well-known ones below: National Hemophilia Foundation The NHF started in 1948, with the goals of discovering better treatments…

Hemophilia is a genetic disorder that affects your blood’s ability to clot. Hemophilia sufferers lack a clotting factor, meaning they have an increased risk of bruising and bleeding, both externally and internally around joints and in the brain. MORE: Learn more about hemophilia treatment options. There are three levels of…

Pharmaceutical firm uniQure N.V. has developed an optimized large-scale process to manufacture its lead gene, therapydate, to treat hemophilia B. This new platform will allow the Dutch company to boost production of its AMT-060 gene therapy at its Lexington, Massachusetts, facility, in accordance with Good Manufacturing Practices guidelines.