Archive for hemophilia
In a thriving gene therapy program, nursing leadership is the driving force
Gene therapy was made possible by decades of technological advances. But to execute gene therapy at scale? That would not be possible without the foresight, organization, and innovation of nursing leadership at Boston Children’s Hospital. The gene therapy implementation framework they have built has become a gold standard for the field. Founded in 2010, the ... Read More about In a thriving gene therapy program, nursing leadership is the driving force
Hemophilia A gene therapy paves the way for a life with less pain
Matthew Harrington, a 35-year-old oil-field worker from Douglas, Wyoming, knows pain. Born with hemophilia A, a rare condition in which the blood doesn’t clot properly, Matt’s body — both inside and outside — has difficulty stopping bleeding. He has what’s considered the most severe form of hemophilia, characterized by frequent bleeding episodes, particularly in the ... Read More about Hemophilia A gene therapy paves the way for a life with less pain
Accessing hemophilia care: A tale of two countries
When Miguel and Marco Antonio were born in the Philippines, they had a 50 percent chance of having hemophilia, as two of their uncles had the condition. “We were just crossing our fingers that they’d fall in the other 50 percent,” says Jojo, their father. But when Miguel was taking his first steps as ... Read More about Accessing hemophilia care: A tale of two countries
Long-term hemophilia treatment could lie in patients’ own cells
Children (and adults) with hemophilia are slow to form blood clots, so are at constant risk for uncontrolled bleeding. Even when the skin isn’t broken, a fall or a simple toe stub can become a serious medical issue: internal bleeding cause permanent damage to muscles and joints. While regularly replacing the missing or malfunctioning clotting ... Read More about Long-term hemophilia treatment could lie in patients’ own cells
How our children’s rare conditions created our special bond
Twenty years ago, Brad McNamara and Joel Klein became roommates at Northeastern University. Little did they know that years later their friendship would become a vital source of support for themselves and their families, connected by the bond of parenting a child with a rare condition. The families’ medical odyssey began in 2014, when Joel ... Read More about How our children’s rare conditions created our special bond
An education in hemophilia for Colin’s new school
Every morning, one of Colin Bazinsky’s parents puts a needle into his chest to give him an infusion of clotting factor. You wouldn’t guess anything was amiss from the placid look on the 3-year-old’s face as he receives the infusion to treat his hemophilia. His state of calm reflects his parents’ matter-of-fact approach to the ... Read More about An education in hemophilia for Colin’s new school
Breaking gender biases: What is it like to be a girl with severe hemophilia?
In human biology class this past semester, Morgan DiPrima viewed a PowerPoint presentation that made her head spin. It included a hemophilia slide solely focused on men. “So, I asked the professor if I could do a class presentation on hemophilia for extra credit.” Very few college students “request” extra work, but this 19-year-old Dean ... Read More about Breaking gender biases: What is it like to be a girl with severe hemophilia?
Taking life with hemophilia one day at a time
Hemophilia has always been part of Kayla Klein’s life. Her father, David, had the condition. Her son, Robbie, has it too. For years, though, Kayla has also surrounded herself with the right people — people who know hemophilia and who have helped her and her husband Joel create a life where the condition isn’t something ... Read More about Taking life with hemophilia one day at a time