A promising step toward safer, less toxic stem cell transplants
Stem cell transplantation (also called bone marrow transplantation) and gene therapy are among the most powerful treatment approaches for blood diseases such as sickle cell disease, beta thalassemia, immune deficiencies, and some blood cancers. Replacing or correcting the blood-forming stem cells can offer the possibility of long-lasting benefit or a cure. However, before patients can ... Read More about A promising step toward safer, less toxic stem cell transplants
Testosterone’s role in gut motility could open the door to targeting treatments for IBS and more
Irritable bowel syndrome (IBS) and other motility disorders can be challenging to treat, with current therapies focused mainly on easing symptoms. Now, recent research led by Meenakshi Rao, MD, PhD, a gastroenterologist in Boston Children’s Division of Gastroenterology, Hepatology and Nutrition, has identified a remarkable way that gut microbes and sex hormones regulate gut motility. The ... Read More about Testosterone’s role in gut motility could open the door to targeting treatments for IBS and more
Beyond average-based medicine: HIE as a blueprint for data-informed care
Historically, outcome prediction in medicine has followed a familiar formula: run a clinical trial, publish the results, guide care based on averages. The model has served for decades, despite its limits. In neonatal care, where decisions can carry lifelong consequences, averages are often insufficient. That’s why Ellen Grant, MD, MSc, director of the Fetal Neonatal ... Read More about Beyond average-based medicine: HIE as a blueprint for data-informed care
Nanoparticle drug combo treats venous malformations
Venous malformations are abnormally shaped veins that develop when the cells lining the blood vessels grow too fast when they aren’t supposed to. Children can be born with them, and as the child grows, the venous malformations can get bigger. Venous malformations are a type of vascular malformation that can range from small and superficial ... Read More about Nanoparticle drug combo treats venous malformations
Biventricular repair linked to better outcomes in superior-inferior ventricles treatment
The complexity of superior-inferior ventricles — a condition in which one ventricle is positioned on top of the other — has often limited surgical treatment. But Boston Children’s heart specialists recognized an opportunity to make treatment for the rare condition more accessible to children. Their solution has been a procedure the hospital is at the ... Read More about Biventricular repair linked to better outcomes in superior-inferior ventricles treatment
Mapping ‘neighborhoods’ in aggressive childhood brain tumors
The third-most common kind of childhood brain tumor, supratentorial ependymoma, is an aggressive cancer that often returns after surgery and radiation therapy. By learning more about the makeup of these tumors, Boston Children’s researchers hope to better understand how to treat them and prevent recurrence. In a new study published in Nature, and led by ... Read More about Mapping ‘neighborhoods’ in aggressive childhood brain tumors