Research
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
Patient Stories
Stay chill, show up, move forward: Nolan’s journey with ITP
At 18 years old, Nolan Ryan is an all-around athlete. Between basketball, football, and lacrosse, sports have shaped his daily life for years. He’s a true team player who shows up to every game with humility and dedication, whether he’s on the field or sitting on the sidelines. Like most student-athletes, Nolan had to balance ... Read More about Stay chill, show up, move forward: Nolan’s journey with ITP
Research
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
Research
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
Patient Stories
Small gains with big meaning: Loic’s story of deep brain stimulation for PKAN care
Loic, 5, lives in Montana with his parents, Sherine and Mark, and his sister, Selah. He loves Spider-Man, sushi, and the Fourth of July. He laughs easily, sings along to his favorite shows, and is a big flirt. These are the little things that Sherine says make Loic, Loic, and which, until earlier this year, ... Read More about Small gains with big meaning: Loic’s story of deep brain stimulation for PKAN care
Patient Stories
‘So much has changed’: Advances in XLH care help siblings thrive
Learn more about our Skeletal Health Center. When Stephanie Capone was a child, she was diagnosed with X-linked hypophosphatemia (XLH), a rare genetic condition that makes bones weak, soft, and easy to fracture. XLH, which can be passed down through families, can lead to difficulty walking, bowlegs, short stature, and other challenges. “I underwent ... Read More about ‘So much has changed’: Advances in XLH care help siblings thrive
Research
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
Research
Gene therapy for hearing loss: Tag-teaming from the lab to the clinic
Two-year-old Miles is one of the first patients with hereditary hearing loss to receive gene therapy at Boston Children’s Hospital — to striking effect. In May 2025, Miles received an injection delivering a healthy copy of a gene called OTOF into the cochlea of his inner ear. Now, Miles is thriving. He’s had no adverse ... Read More about Gene therapy for hearing loss: Tag-teaming from the lab to the clinic
Patient Stories
A rare injury, a more determined player: Adrian’s knee dislocation
Adrian can barely remember a time when he didn’t play football. Now 16, he grew up watching games with his father, Antron, who’d played for a top-tier college and practiced with the NFL. In addition to televised games, the two often traveled to Tennessee for football weekends with his father’s former teammates. “Football is a ... Read More about A rare injury, a more determined player: Adrian’s knee dislocation
Patient Stories
A reunion of the heart: Grace and Amy bond through HLHS
At first glance, Grace and Amy have a pretty typical friendship: they joke around, cheer each other on, and keep in touch mostly through texts and FaceTime. But look a little deeper and there’s something extraordinary about their bond, or more specifically, how and where it began. Grace, 22, and Amy, 21, were both born ... Read More about A reunion of the heart: Grace and Amy bond through HLHS