Spark Therapeutics will present novel results regarding the ongoing Phase 1/2 clinical trial (NCT02484092) investigating SPK-9001 in hemophilia B patients at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, to be held July 8-13 in Berlin. Two presentations focus on data from the trial: “SPK-9001:…
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A new study suggests that whole body vibration training increases muscle strength, bone mineral density, and functional capacity in children with hemophilia. The study, “Effect of whole body vibration training on quadriceps strength, bone mineral density, and functional capacity in children with hemophilia: a randomized clinical trial,” was published…
Hemophilia is a blood disorder that’s usually genetically inherited from parents. People born with hemophilia have little or no clotting factor – a protein needed for normal blood clotting. Hemophilia can also be acquired, meaning patients don’t inherit the disease from their parents but instead develop it during their lifetime. This…
An international team has joined efforts to establish guidelines for effectiveness and outcome measurements regarding gene therapies in hemophilia. The CoreHEM project will be led by researchers from McMaster University in Ontario, Canada, in collaboration with the National Hemophilia Foundation (NHF) in the U.S. and the Green Park…
3 Tips for Coping With a Hemophilia Diagnosis
Discovering your child has hemophilia can be devastating. While you will certainly need to make some lifestyle changes moving forward, with the correct treatment and preventative measures, there’s no reason why your child can’t live a happy and healthy life. To help you come to terms with life with hemophilia and…
The advancements in modern medicine have meant that people with hemophilia are living longer than ever, however, this brings its own set of problems for the medical profession. People living with the disease are just as likely to have other health issues when they get older as those without hemophilia,…
How Kids Can Play Safe With Hemophilia
In this video from the Centers for Disease Control and Prevention, learn how children with hemophilia can play and live safely. The simple animation features four boys who all have hemophilia but love to play sports. MORE: How hemophilia is inherited. The film advises that…
13 Fast Facts About Hemophilia
Hemophilia is a rare inherited disease where a person is lacking a certain type of clotting factor. This means that their blood is unable to clot so injuries will result in extensive bleeding. If left untreated, this bleeding can become life-threatening. Here are a few fast facts about this rare…
FDA Accepts Bioverativ’s Investigational New Drug Application for BIVV001 to Treat Hemophilia A
The U.S. Food and Drug Administration (FDA) has accepted Bioverativ’s investigational new drug application (IND) for BIVV001, an investigational Factor VIII therapy designed to extend protection from bleeds in hemophilia A patients. The FDA will now review the IND application for safety. The Waltham, Massachusetts-based company plans to initiate a Phase…
What Is Acquired Hemophilia?
The majority of hemophilia cases are genetic, affect males and are diagnosed fairly early in childhood. However, there are rare cases of hemophilia that develop later in life and are called “acquired hemophilia.” MORE: Four hemophilia screening tests. Acquired hemophilia is an autoimmune disease where the immune system attacks blood clotting…