Which children in status epilepticus are most at risk?
Status epilepticus, a prolonged, potentially life-threatening seizure, is epilepsy’s most severe manifestation. Patients known to have refractory status epilepticus are more likely to need ICU care if initial treatment is delayed. But what about the broader population of children who come to the emergency room in status epilepticus? Are there any early indicators to predict ... Read More about Which children in status epilepticus are most at risk?
Zika study reveals how infection can cause microcephaly
Prenatal exposure to viruses capable of infecting the fetal brain, particularly in the first trimester, can cause a range of developmental defects in the baby. The Zika epidemic in Brazil during 2015-2016 posed an extreme case, causing hundreds of babies to be born with microcephaly, or an abnormally small head. Although cases have waned significantly, ... Read More about Zika study reveals how infection can cause microcephaly
Studies pinpoint risk factors for complications of pediatric intestinal failure
Pediatric intestinal failure occurs when patients lack the amount of intestine necessary for growth and development. As advances in treatment have led to better survival rates for children with short bowel syndrome and other forms of intestinal failure, the team at Boston Children’s Center for Advanced Intestinal Rehabilitation (CAIR) is studying factors that affect quality ... Read More about Studies pinpoint risk factors for complications of pediatric intestinal failure
Tackling an aggressive, treatment-resistant lymphoma where it lives
Anaplastic large cell lymphoma, a form of non-Hodgkin lymphoma, is the most common aggressive lymphoma in children. Chemotherapy and radiation fail to cure about 30 percent of cases. When tumors are driven by the oncogene ALK — which is the case for the majority of children — kinase inhibitor drugs like crizotinib are very effective ... Read More about Tackling an aggressive, treatment-resistant lymphoma where it lives
One-time treatment could block a deadly form of graft-versus-host disease
Even when a bone marrow transplant cures leukemia or lymphoma, patients can still pass away from graft-versus-host disease (GVHD), in which T cells in the donor graft attack the recipient’s own tissues. Leslie Kean, MD, PhD, director of stem cell transplant at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, has long sought to prevent this ... Read More about One-time treatment could block a deadly form of graft-versus-host disease
From bench to bedside: A promising option for unremitting ulcerative colitis
Many existing treatments for inflammatory bowel disease, like Remicade® and Humira®, work by blocking inflammatory cytokines such as tumor necrosis factor (TNF) and interleukins IL-12 and IL-23, which are over-produced in autoimmune disease. But few patients with ulcerative colitis get complete relief from these drugs. A small but promising open-label clinical trial, published recently in ... Read More about From bench to bedside: A promising option for unremitting ulcerative colitis
Perfecting the craft of modeling disease in stem cells: Dosh Whye
Part of an ongoing series profiling researchers at Boston Children’s Hospital. Dosh Whye has always wanted to make peoples’ lives better, but he never imagined that tending to stem cells in a lab seven days a week would be the way he would do it. Now, as an assistant director of the Human Neuron Core ... Read More about Perfecting the craft of modeling disease in stem cells: Dosh Whye
Seeking a path forward for custom genetic treatments
In 2018, at age 7, Mila Makovec became the world’s first person to be treated with a drug made just for her. Languishing from Batten disease, a rare, fatal neurodegenerative disorder, she received an antisense oligonucleotide (ASO) drug designed to silence her genetic mutation, injected into the fluid around her spine through a lumbar puncture. ... Read More about Seeking a path forward for custom genetic treatments
Facial weakness: A dark matter detective story
Elizabeth Engle, MD, has devoted her career to finding genetic and developmental causes for disorders of eye, eyelid, and facial movement. From common conditions like strabismus to very rare disorders, these conditions can impact a person’s appearance and impair social communication, making it hard to shift one’s eyes up, down, or sideways or adjust facial expressions. Each ... Read More about Facial weakness: A dark matter detective story
Finding a way to help newborns who can’t immediately have heart treatment
Newborns with complex congenital heart defects (CHD) and pulmonary overcirculation often need treatment as soon as possible. Unfortunately, some of these children are not in good enough health to withstand surgery. To address this challenge, Boston Children’s heart specialists leaned into technological innovation, their experience, and a perseverance that would ultimately confirm their belief that even the ... Read More about Finding a way to help newborns who can’t immediately have heart treatment