While tremendous advancements have been made over the past several decades in treating cystic fibrosis (CF), many CF therapies are not one-size-fits-all. What works for adult patients doesn’t necessarily help very young patients. And yet, it’s critical to begin therapies early in life to delay lung disease caused by CF from progressing.
Inhaled hypertonic (extra salty) saline is one such therapy recommended for many CF patients age 6 or older, but its effectiveness has never been evaluated in patients age 5 or younger. Despite this, since 2007 inhaled hypertonic saline has been increasingly used among U.S. children with CF ages 2 to 5.
Several years ago, Australian researchers stumbled on the benefits of hypertonic saline when they noticed that surfers with CF had fewer respiratory flare-ups than people with CF who didn’t surf. The researchers speculated that the salty mist of ocean water lessened respiratory CF symptoms and their subsequent study confirmed their hypothesis.
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Sometimes even a simple idea can make a difference in people’s lives. In my case, I hope that the creative adaptation of a simple science concept will one day save the lives of premature infants in the developing world.
As a program manager in the Center for Developmental Therapeutics, I support researchers working in the realm of preterm birth and neonatal health. So far, the main focus of that work has been on treatment options for premature infants, including the development of the Seattle Children’s Positive Airway Pressure ( Sea-PAP) device, an easy to use respiratory support device for preemies.
The aim of my research project is to investigate new ways of easily and inexpensively diagnosing lung immaturity in premature infants. Premature birth is a leading cause of neonatal death in the U.S., but not many people realize that it is also a huge issue in developing countries. New research has shown that 15 million premature babies are born each year, and helping preterm infants everywhere lead long, healthy lives is an important goal of my research. Read full post »
“Baby born at 1 pound now a thriving 5-year-old” “Barely over a pound when born, miracle baby will go home” These are just two of the headlines that have crossed the wires in the past week. And while they convey the hope and possibilities now available to babies born too soon in the developed world, they fail to paint an accurate picture of premature birth.
Premature birth has become the second leading cause of death in children under the age of five, killing 1.1 million infants worldwide. In the United States and other developed countries, medical interventions are able to save many preterm babies, resulting in the “miracle baby” stories we see each week. The story that’s rarely reported is that these babies often face a lifetime of disability that may include cerebral palsy, brain injury, or respiratory, vision, hearing, learning, and developmental problems. The impact on families is huge; economic turmoil from medical bills, lost wages if a parent is needed full-time at-home to address the medical challenges, and emotional strain from managing the situation. Read full post »
Dr. Bonnie Ramsey of Seattle Children’s Research Institute was honored today, April 18, in Washington, D.C. for her work on clinical trials of Kalydeco, a cystic fibrosis drug approved by the U.S. Food and Drug Administration (FDA) this year. She is a co-recipient of the award with Dr. Frank Accurso from the University of Colorado.
The Clinical Research Forum, a nonprofit organization that promotes understanding and support for clinical research and its impact on health and healthcare, put Dr. Ramsey’s work on a Top Ten list of outstanding clinical research projects across the country. Some of Dr. Ramsey’s fellow award winners were behind research that: is helping prevent complications after bone marrow transplantation; uncovered new therapies for leukemia; found that early treatment with medications can prevent HIV transmission. Read full post »
Like many parents across the country, my husband and I are constantly trying to balance and prioritize what’s best for our two young children. I’m also a researcher at Seattle Children’s Research Institute and a pediatrician at Seattle Children’s Hospital and the University of Washington. My most recently-published work highlights that half of preschool age children in the U.S. lack daily outdoor playtime with their parents. The topic is one that hits close to home, as there are many challenges for parents to get outside with their little ones. Read full post »