Research
Could a pill treat sickle cell disease?
The new gene therapies for sickle cell disease — including the gene-editing treatment Casgevy, based on research at Boston Children’s Hospital — have been game-changing for the patients who have received them. But Stuart Orkin, MD, the Boston Children’s hematologist whose work led the way to Casgevy, wants to go even further. “The editing therapy ... Read More about Could a pill treat sickle cell disease?
Research
A sickle cell first: Base editing, a new form of gene therapy, leaves Branden feeling ‘more than fine’
Though he doesn’t remember it, Branden Baptiste had his first sickle cell crisis at age 2. Through elementary school, he was in and out of the hospital with pain episodes, not knowing why. As he got older, he learned he had sickle cell disease. His red blood cells were forming sickle shapes and getting stuck ... Read More about A sickle cell first: Base editing, a new form of gene therapy, leaves Branden feeling ‘more than fine’
Health and Parenting
Keeping up with the tween skin care trend: What’s in those products?
More teens and tweens are hitting the mall to grab skin-care products they’ve seen on social media — but are these popular ingredients safe for younger skin? We asked Dr. Jennifer Tan and Dr. Jennifer Huang in Boston Children’s Dermatology Program to weigh in on this trend and break down common ingredients. Trends aren’t for ... Read More about Keeping up with the tween skin care trend: What’s in those products?
Patient Stories
Then and now: Catching up with some of our short bowel syndrome kids
Also known as “short gut,” short bowel syndrome is a life-threatening condition in which a lack of functioning intestine can make it difficult to absorb the nutrients needed to survive and thrive. Thanks to advances in medical and surgical treatments — many of which have been made by the team in Boston Children’s Center for Advanced ... Read More about Then and now: Catching up with some of our short bowel syndrome kids
Research
Injected microbubbles could be a safe way to deliver emergency oxygen
For years, researchers and clinicians have been trying to find a way to rapidly deliver oxygen to patients when traditional means of oxygenation are difficult or ineffective during critical moments of cardiac or respiratory arrest. Sometimes, hypoxemia caused by airway obstruction or lung disease can be so severe that methods to boost low-oxygen levels (including ... Read More about Injected microbubbles could be a safe way to deliver emergency oxygen
Research
A universal gene therapy for Diamond-Blackfan anemia is poised for clinical testing
Diamond-Blackfan anemia (DBA), first described at Boston Children’s Hospital in 1938, is a rare blood disorder in which the bone marrow cannot make mature, functioning red blood cells. Children with this life-threatening anemia have few treatment options. A small handful with a well-matched donor can be cured with bone marrow transplant, but most rely on ... Read More about A universal gene therapy for Diamond-Blackfan anemia is poised for clinical testing
Patient Stories
A heavy-medaled gymnast and his close call with leg-length discrepancy
Ask 15-year-old Kaleb what he likes about gymnastics and with a sly smile, he’ll say, “flipping around.” That’s Kaleb: understated, funny. But watch him in action, and you’ll see a focused gymnast who earned two medals in the 2024 Men’s Eastern National Championships. He probably won’t mention that two years ago, a surgeon told his ... Read More about A heavy-medaled gymnast and his close call with leg-length discrepancy
Research
Skin organoid could guide new treatments for skin conditions, hair loss
What does it take to build healthy skin? Two research groups converged on this question from different angles. They’ve now produced the most detailed view to date of the cell types and cell collaborations that go into creating our body’s largest organ. Several years ago, Karl Koehler, PhD, and colleagues at Boston Children’s Hospital used ... Read More about Skin organoid could guide new treatments for skin conditions, hair loss
Research
Fuel to be faster: Studying the effects of low energy availability at the Boston Marathon
Like many sports medicine specialists, Kristin Whitney, MD, MA, suspected that many of the issues she treats in runners — bone stress injuries, anemia, decreased response to training, and reduced endurance to name a few — stemmed from insufficient nutrition. Key takeaways Runners with indicators of low energy availability had slower finish times and more ... Read More about Fuel to be faster: Studying the effects of low energy availability at the Boston Marathon
Patient Stories
For Fiadh: Pushing the envelope to treat drug-resistant epilepsy
Fiadh is a bubbly and empathetic 4-year-old. And if you ask her parents, Elaine and Dario, she’s also mischievous and wild. “She knows when she shouldn’t be doing something,” Elaine says. “Then she gives you a look, giggles, and does it anyway.” Fiadh’s tenacity likely comes from her mother, which is a good thing. After ... Read More about For Fiadh: Pushing the envelope to treat drug-resistant epilepsy