Service dogs are typically thought of as necessary companions for the visually impaired, but service and therapy dogs can be a practical solution for people with a variety of chronic illnesses. As well as being a trusted friend, service dogs can expand owners’ motor abilities, granting them new independence and allowing them…
Dimension Therapeutics will discontinue clinical development of DTX101, a gene therapy intended to treat moderate/severe-to-severe hemophilia B. The decision comes after a review of data from a Phase 1/2 clinical trial (NCT02618915), which assessed and the safety and dosing of DTX101. The trial was a…
The first recorded mention of hemophilia came in the second century when a collection of ancient Jewish writings noted that baby boys did not need to be circumcised if two of their older brothers had bled to death following the procedure. Later in the 10th century, an Arabian doctor described cases of…
Sangamo Therapeutics and Pfizer have entered a global collaboration and license agreement to develop and market gene therapy programs for hemophilia A, including SB-525. SB-525 was cleared by the U.S. Food and Drug Administration (FDA) in January as an investigational new drug (IND) for the…
Hemophilia is a rare blood disorder that primarily affects males, but females can be carriers of the disease. There are two different types of hemophilia carriers: possible carriers and obligate carriers. According to Steps For Living, the mutated genes associated with hemophilia lie on the X chromosome. If a woman…
A new online service allows patients with bleeding disorders like hemophilia to share important information about their treatment and bleeds with their healthcare team in real time. MORE: Five tips for living healthily with hemophilia. The HemaGo XChange portal was created by Novo Nordisk A/S as an update to their existing…
UniQure has taken major steps this year toward U.S. and European regulatory approval of AMT-060, its gene-therapy treatment for hemophilia B. In January, the U.S. Food and Drug Administration (FDA) gave AMT-060 breakthrough therapy designation for patients with severe hemophilia B. In April the European Medicines Agency (EMA) gave it PRIME designation. The designations…
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…
Acquired hemophilia is an autoimmune disorder in which the immune system mistakes proteins that are part of the body as foreign and starts producing antibodies against them. The disease shares few similarities with inherited hemophilia, a bleeding disorder that affects mostly males from birth. Acquired hemophilia, a very rare condition…
Hemophilia is a disease where a person blood lacks a factor which enables it to clot. This can lead to severe bleeding which can be life-threatening if left untreated. According to the World Federation of Hemophilia, there are three types of hemophilia: inherited, sporadic, and acquired. MORE: Five…
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