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‘Bubble Boy’ Disease, Nearly Always Fatal, Could Have a Cure

December 12, 2013
An international consortium of scientists, including researchers at Dana-Farber/Boston Children's, is finding promising early results in a new gene therapy clinical trial they say may lead to an eventual long-term cure for SCID-X1, ABC News reports.

‘It’s not an even playing field’

January 30, 2017
An emerging area of research explores the impact of poverty and financial hardship on care and outcomes for children with cancer, WBUR reports.

“Stapled” antimicrobial peptides could combat antibiotic resistance

August 19, 2019
The peptides, folded into specific three-dimensional shapes using chemical “staples” for reinforcement, kill multidrug resistant bacteria but don’t harm red blood cells or other normal tissues.

2020 Harrington Prize awarded to Dr. Stuart H. Orkin

April 16, 2020
The Harrington Discovery Institute and The American Society for Clinical Investigation honor Dr. Orkin for his contributions to red blood cell biology.

A bold strategy to enhance CAR T-cell therapies, capable of targeting DIPG and other tough-to-treat cancers

April 27, 2018
As reported in Vector, a Boston-based team of researchers, made up of scientists and pediatric oncologists, believe a better CAR T-cell therapy is on the horizon.

A breakthrough in our understanding of how red blood cells develop

March 15, 2018
In this Vector report, scientists make a new discovery about what drives the development of mature red blood cells after taking a deep dive into the molecular underpinnings of Diamond-Blackfan anemia.

A Child Saved Becomes a Teen Hunting for Cures

September 08, 2014
Fourteen years ago, Loren Walensky, MD, PhD, diagnosed Kate Franklin with leukemia. Over the summer Kate, now a 17-year-old aspiring doctor, was an intern in the lab of the doctor who saved her life, The Boston Globe reports.

A new gene therapy transplantation technique could improve treatment of neurodegenerative disease

December 06, 2017
Alessandra Biffi, MD, led a study that shows the efficacy of a new gene therapy transplantation technique that could have future applications for treating neurodegenerative diseases.

A new, faster route for pediatric clinical trials

July 13, 2010
Researchers hoping to test promising cancer therapies for children now have an easier way to navigate the forest of funding applications, regulatory rules, hiring procedures, review boards, statistical services, and other logistical requirements.

A Young Cancer Patient’s Take on “The Fault in Our Stars”

June 21, 2014
Anya Harp, a 13-year-old leukemia patient, saw the hit movie “The Fault in Our Stars” and describes her reaction to it in The Boston Globe.
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