Blog | Page 12 of 158 | Boston Children's Answers

Mapping cells to create targeted treatments for interstitial lung disease

Posted on January 13, 2025 by Albert McKeon | Basic/Translational, Research

Tags: genetics and genomics, interstitial lung disease, precision medicine, pulmonology, research

John Kennedy, MD, MSc, remembers the relative simplicity of his first genetic mapping project. In a Harvard Medical School lab, he helped map a gene for the neurological disease mucolipidosis type IV in less than a year. “I was fresh out of college. I thought with the global momentum of the Human Genome Project, we were going to ... Read More

Tagged: genetics and genomics, interstitial lung disease, precision medicine, pulmonology, research

Faced with a rare type of liver cancer, Ramie focuses on the positive

Posted on January 6, 2025 by Jessica Cerretani | Patient Stories

Tags: liver cancer, transplant

When Ramie Darling learned that his liver cancer had recurred, he thought for a while and then simply said, “OK.” Asked by his parents if he understood the situation, he replied, “I know I need more treatment. But whenever there’s a ‘down’ in life, I just think of the ‘ups.’” It’s an attitude that has ... Read More

Tagged: liver cancer, transplant

Blood across our lifetimes: An age-specific ‘atlas’ tells a dynamic story

Posted on January 3, 2025 by Nancy Fliesler | Basic/Translational, Research

Tags: blood, blood disorder, cancer, leukemia, stem cells

The stem cells that form our blood, also known as hematopoietic stem cells (HSCs), are with us throughout our lives. A new study reveals how HSCs ramp up and pivot their activities depending on the body’s needs at the time, from before we’re born until old age. Researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders ... Read More

Tagged: blood, blood disorder, cancer, leukemia, stem cells

AI-enabled medical devices are burgeoning, but many haven’t been tested in children

Posted on December 18, 2024 by Nancy Fliesler | Data Science, Research

Tags: artificial intelligence, cardiology, clinical trials, imaging, medical devices, neurology

Medical devices that incorporate artificial intelligence and machine learning are proliferating. In 2013, the FDA approved fewer than 10 such devices; by 2023, this number had grown to nearly 250. Devices include software to help interpret radiology images, wearable devices that monitor the heart and flag risks, and devices that analyze brain signals for potential ... Read More

Tagged: artificial intelligence, cardiology, clinical trials, imaging, medical devices, neurology

Years of experience support the safe use of fine-needle aspiration for pediatric thyroid nodules

Posted on December 17, 2024 by Jessica Cerretani | Research

Tags: thyroid cancer

Ultrasound-guided fine-needle aspiration (FNA) has long been used as a safe and effective way to evaluate thyroid nodules in adults, usually without sedation. Less is known about the safety and tolerability of FNA in children, although small studies suggest that the approach can be performed without sedation and with minimal complications. Now, a report by ... Read More

Tagged: thyroid cancer

A small act of kindness: Blood donations get Sadie off the sidelines after her aplastic anemia diagnosis

Posted on December 16, 2024 by Linsey Covino-Deaso | Patient Stories

Tags: anemia, blood disorder, blood donor center, rare disease

In March of 2024, Sadie’s life was interrupted. A busy high school senior with classes to attend, soccer matches to play, and a much-anticipated trip to France on the horizon, Sadie was very active and felt generally healthy. But when a follow-up appointment at her pediatrician’s office led to an urgent referral to Boston Children’s ... Read More

Tagged: anemia, blood disorder, blood donor center, rare disease

Changing lives through genetics: The Children’s Rare Disease Collaborative

Posted on December 13, 2024 by Nancy Fliesler | Clinical Care, Research

Tags: diagnostics, genetics and genomics, rare disease, research

A 14-year-old girl was having back pain after a car accident and visited an orthopedic clinic at Boston Children’s Hospital. In the course of her care, she joined the Children’s Rare Disease Collaborative (CRDC), a hospital-wide effort to enroll children and adults with rare diseases in genetic studies. Genetic testing revealed that both she and ... Read More

Tagged: diagnostics, genetics and genomics, rare disease, research

Model enables study of age-specific responses to COVID mRNA vaccines in a dish

Posted on December 12, 2024 by Nancy Fliesler | Clinical, Research

Tags: coronavirus, immunology, infectious diseases, vaccines

mRNA vaccines clearly saved lives during the COVID-19 pandemic, but several studies suggest that older people had a somewhat reduced immune response to the vaccines when compared with younger adults. Why? Researchers at Boston Children’s Hospital, led by Byron Brook, PhD, David Dowling, PhD, and Ofer Levy, MD, PhD, found some answers — while providing ... Read More

Tagged: coronavirus, immunology, infectious diseases, vaccines

Could a pill treat sickle cell disease?

Posted on December 11, 2024 by Nancy Fliesler | Basic/Translational, Research

Tags: drug development, hematology, 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

Tagged: drug development, hematology, sickle cell disease

A sickle cell first: Base editing, a new form of gene therapy, leaves Branden feeling ‘more than fine’

Posted on December 9, 2024 by Nancy Fliesler | Clinical, Research

Tags: clinical trials, gene editing, gene therapy, hematology, sickle cell disease

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

Tagged: clinical trials, gene editing, gene therapy, hematology, sickle cell disease