Cell therapies may be a feasible way of achieving a long-term correction of bleeding disorders, preclinical data shared by Sigilon Therapeutics show. A single implant of Sigilon’s engineered cells given to mice enabled durable production of therapeutic levels of blood clotting factors and normalized bleeding time. The technology uses the…
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Severe, moderate, and mild: These are the degrees with which a person with hemophilia is labeled based on the percentage of clotting factor in their blood. With the word “severe,” one might automatically think it is the worst kind of hemophilia. This level means that a person has less…
I’m happy to finally be an official member of the Father’s Day club. I’ve learned much from the way my own family raised me. It’s an uphill battle raising our daughter Cittie with the occasional bleed dragging me down. But seeing her mature so quickly…
Twenty-three years ago, my life changed dramatically. I became a mom. My firstborn, Julian, entered the world screaming at the top of his lungs with a head full of crazy black hair. I imagined him thinking, “Put me back in!” When he was laid on my chest, I was overcome…
I recently had the opportunity to spend the afternoon with a group of teens from our local bleeding disorders organization, HACA, which stands for Hemophilia Association of the Capital Area. Some of these kids have gone to camp together for years, and some were brand new to the group.
The month of May can be an emotional roller coaster for many. Social media fills up with parents bragging about their children’s latest accomplishments — honor society, college acceptance letters, awards, and recognitions among them. It’s a great thing to have a network for connection and celebration of each…
I am a patient. I am an expert on my own health experiences. I am an educator. I am an advocate. Hemophilia is a rare disease. Being a woman with a disorder thought once to be male-only makes it complicated to navigate. I…
ASC Therapeutics has gotten exclusive rights to Expression Therapeutics‘ technology to develop liver-targeting hemophilia A gene therapies. “We have a common mission with Expression Therapeutics — to develop novel therapies that will dramatically improve the lives of persons with hemophilia around the world. Hemophilia A is a…
Hemophilia is a blood disorder in which patients do not make enough of the clotting factors that control bleeding. Following accident or injury, people with hemophilia may be unable to stop bleeding. In severe cases, patients may bleed spontaneously at joints. While there is no cure for hemophilia, there…
Replacement therapy is one of the standard therapies used to treat hemophilia, a genetic disorder caused by the absence or defects in blood clotting factors. Hemophilia makes patients highly susceptible to bleeding due to a compromised cascade of clotting reactions that are necessary to “seal” injuries.