Archive for blood
PK deficiency clinical trial: Jim’s volleyball ace
Growing up with pyruvate kinase deficiency (PK deficiency) wasn’t always the easiest thing, but it was my reality. My visits to Dana-Farber/Boston Children’s Cancer and Blood Disorders Center helped give me perspective — although my condition was difficult to deal with, it certainly was not the worst condition I could have. It made me grateful ... Read More about PK deficiency clinical trial: Jim’s volleyball ace
Too many blood cells: Probing a blood cancer’s genetic origins
At age 7, Meredith Shah began having debilitating migraine headaches. “I would have trouble seeing and shapes were blurry,” she recalls. “It was really painful.” Over time, the frequency and intensity of the headaches escalated. Her parents, Heidi and Nil, sought the help of multiple specialists. But they received few answers, other than an indication ... Read More about Too many blood cells: Probing a blood cancer’s genetic origins
Platelet profiles could enhance care for bleeding disorders, cardiovascular disease, cancer
The trillion-odd platelets in our blood are best known for controlling bleeding, helping clots form when we sustain an injury. But platelets can also go rogue. They contribute to clotting in cardiovascular disease and diabetes, leading to strokes and heart attacks, and can be hijacked by cancers to help them spread. Key takeaways A new ... Read More about Platelet profiles could enhance care for bleeding disorders, cardiovascular disease, cancer
Trial tests dornase alfa, a cystic fibrosis drug, for severe COVID pneumonia
Boston Children’s Hospital and Brigham and Women’s Hospital will soon begin testing an existing drug, dornase alfa, in patients with severe COVID-19 pneumonia and respiratory failure. The randomized, controlled clinical trial aims to enroll 60 adults and children over age 3 who require mechanical ventilation. Key takeaways:· A randomized trial is testing whether a cystic ... Read More about Trial tests dornase alfa, a cystic fibrosis drug, for severe COVID pneumonia
Tracking an organism’s development, cell by cell
A new mouse model allows scientists to track every cell in the body — from the embryo stage until adulthood. The system is the first of its kind and should yield a greater understanding of development, aging, and disease. Scientists described it last week in the journal Cell. “The dream of many developmental biologists for ... Read More about Tracking an organism’s development, cell by cell
A drug treatment for telomere diseases?
For years, Donna Martin carried a piece of scrap paper with the words “dyskeratosis congenita,” which she believed might explain her son Brad’s sudden, mysterious affliction. A routine blood test had revealed Brad’s bone marrow was failing, unable to keep up with his need for healthy blood cells. His condition, Donna knew, would worsen over ... Read More about A drug treatment for telomere diseases?
Following blood stem cells in the body could yield better, safer treatments
Blood stem cells make all the different kinds of blood and immune cells in our body. Scientists have long been trying to make these cells in the lab and use them to make different blood cell types on demand. Mastering these feats could help make bone marrow transplants more efficient and less toxic, and could ... Read More about Following blood stem cells in the body could yield better, safer treatments
Going into science: Women scientists at Boston Children’s offer advice to girls
In honor of the International Day of Women and Girls in Science (February 11), we invited women scientists at all stages of their careers at Boston Children’s Hospital to share their scientific agendas. Here is some of what they had to say. The scientists also offered their advice for girls interested in entering the field. ... Read More about Going into science: Women scientists at Boston Children’s offer advice to girls
Scarlette: Hoping for a bright future with PK deficiency
Two-year-old Scarlette Walker loves pink. For her January visit to the Hematology Clinic at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, she wore a shocking pink hat and pink dress. “She picked them out herself,” says her mom, Olivia. She even has a pink alter ego. “She pretends she is a pink helicopter named Dizzy. If ... Read More about Scarlette: Hoping for a bright future with PK deficiency
Good early results with gene therapy for rare immune deficiency
Brenden Whittaker, a college student in Ohio, has been caught off guard by his good health. Since he was young, a rare immune deficiency known as chronic granulomatous disease (CGD) had left him vulnerable to life-threatening infections. He was used to going in and out of the hospital, and then hooking up to an IV ... Read More about Good early results with gene therapy for rare immune deficiency