Microvillus inclusion disease: From organoids to new treatments
Microvillus inclusion disease (MVID) is a rare type of congenital enteropathy in infants that causes devastating diarrhea and an inability to absorb food. Infants can lose liters of fluid a day, become severely dehydrated, and stop growing. There is no specific treatment. “Until about 10 years ago, 50 percent of kids with MVID would die ... Read More about Microvillus inclusion disease: From organoids to new treatments
Exposing a tumor’s antigens to enhance immunotherapy
Successful immunotherapy for cancer involves activating a person’s own T cells to attack the tumor. But some tumors have a trick: They hide themselves from the immune system by preventing their antigens from being displayed, a necessary step in activating T cells. In new work published in Science, researchers in the Program in Cellular and ... Read More about Exposing a tumor’s antigens to enhance immunotherapy
Combining CAR-T cells and inhibitor drugs for high-risk neuroblastoma
Chimeric antigen receptor (CAR)-T cell therapy is a potent emerging weapon against cancer, altering patients’ T cells so they can better find and destroy tumor cells. But CAR-T cell therapy doesn’t work well in every cancer — including many cases of neuroblastoma, a cancer that begins in young children’s nerve tissue and can metastasize to ... Read More about Combining CAR-T cells and inhibitor drugs for high-risk neuroblastoma
One day closer: Second opinion for urologic pain changes Iker’s life at last
Like many kids, Iker Guzman enjoys playing with LEGO toys. But there was nothing lighthearted about the day a few years ago when he used the tiny bricks to spell out one word on the floor: HELP. It was a message his parents, Elsa and David, had already taken to heart. For years, they had ... Read More about One day closer: Second opinion for urologic pain changes Iker’s life at last
Rowan the Remarkable: Defying the odds with CPAM
This is the story of a baby named Rowan and his remarkable journey of beating the odds after doctors discovered a potentially fatal mass on his lung in utero. This is also the story of his mother, Casey, who fought for him every step of the way, and the medical professionals whose collaboration and planning ... Read More about Rowan the Remarkable: Defying the odds with CPAM
Twenty years after a groundbreaking biventricular repair, Faith gives back by helping children with CHD
Faith Brackett doesn’t remember every detail of the time she was among the first children to have a new life-saving heart surgery. She was 7, after all, when she had a biventricular repair — a procedure that creates two functioning ventricles in a heart relying on just one. But she does remember how the surgery finally gave ... Read More about Twenty years after a groundbreaking biventricular repair, Faith gives back by helping children with CHD
It takes a village and the world: Tariq’s care for Tourette syndrome
When your child is sick but you can’t figure out the cause or how to fix it, it can leave you feeling helpless and frustrated. It can also test how far you’ll go for answers. Just ask Salem of the United Arab Emirates (UAE), who moved with his family to Boston so his son Tariq ... Read More about It takes a village and the world: Tariq’s care for Tourette syndrome
When diagnosis is just the first step: The Brain Gene Registry
Through advances in genetic sequencing, many children with rare, unidentified neurodevelopmental disorders are finally having their mysteries solved. But are they? “Once families receive results of genetic testing, that’s just the beginning of a new journey,” says Maya Chopra, MBBS, FRACP, an investigator with the Rosamund Stone Zander Translational Neuroscience Center at Boston Children’s Hospital. ... Read More about When diagnosis is just the first step: The Brain Gene Registry
Revisiting race and ethnicity in clinical guidelines
Health care institutions often rely on clinical pathways in assessing patients and making decisions about their care. Some of these care algorithms incorporate race, ethnicity, or ancestry as factors in decision making. But is this helpful or harmful? And is there solid evidence to justify treating people of different racial or ethnic backgrounds differently? Robert ... Read More about Revisiting race and ethnicity in clinical guidelines
Helping clinicians embrace family-centered rounds
If you’ve ever been hospitalized, you may have experienced this: groups of doctors coming in and talking about you like you’re not there or addressing you in a perfunctory manner, using medical jargon you don’t understand. Peggy Markle was taken aback when her son was hospitalized for a not-yet-defined condition in Washington and his care ... Read More about Helping clinicians embrace family-centered rounds