Search Results for: dana-farber boston children's
It’s personal: How the Boston Children’s progeria research community brought new life to an old drug
In late November, the U.S. Food and Drug Administration (FDA) approved a repurposed cancer drug called lonafarnib to treat Hutchinson-Gilford …Read More
Nurses Week 2021: Nurse-led contact tracing team helps reduce the spread of COVID-19
As COVID-19 evolved into a serious threat in March 2020, protecting the health of patients, families, and employees at Boston …Read More
Tough cookie: Steroid therapy helps Alessandra thrive with Diamond-Blackfan anemia
Two-year-old Alessandra is many things. She’s sweet, happy, curious, and, according to her parents, Ralph and Irma, a budding food …Read More
A new druggable cancer target: RNA-binding proteins on the cell surface
In 2021, research led by Ryan Flynn, MD, PhD, and his mentor, Nobel laureate Carolyn Bertozzi, PhD, opened a new …Read More
A small act of kindness: Blood donations get Sadie off the sidelines after her aplastic anemia diagnosis
In March of 2024, Sadie’s life was interrupted. A busy high school senior with classes to attend, soccer matches to …Read More
Could a pill treat sickle cell disease?
The new gene therapies for sickle cell disease — including the gene-editing treatment Casgevy, based on research at Boston Children’s …Read More
Cerebral adrenoleukodystrophy: How the Cooksons dodged a devastating disease
Heather Cookson believes that if she hadn’t insisted her son Ricky get a brain MRI to investigate his frequent headaches, …Read More
Gene therapy for adrenoleukodystrophy: Studies find both risks and benefits
Cerebral adrenoleukodystrophy (CALD), portrayed in the film Lorenzo’s Oil, is a devastating disorder caused by a mutation on the X …Read More
In a thriving gene therapy program, nursing leadership is the driving force
Gene therapy was made possible by decades of technological advances. But to execute gene therapy at scale? That would not …Read More
Blood donations help Kit manage Diamond-Blackfan anemia — so she can dance, sing, and enjoy life
Every month, Kit Murdoch needs a blood transfusion to stay alive. The 2-year-old has Diamond-Blackfan anemia, a rare genetic blood disorder …Read More