Patient Stories
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 progeria syndrome, better known as progeria — an ultra-rare incurable genetic disease associated with rapid aging. On average, children with progeria die before age 15, usually the result of rapidly accelerated atherosclerosis resulting in heart ... Read More about It’s personal: How the Boston Children’s progeria research community brought new life to an old drug
Clinical Care
After decades of evolution, gene therapy arrives
As early as the 1960s, scientists speculated that DNA sequences could be introduced into patients’ cells to cure genetic disorders. In the early 1980s, David Williams, MD, and David Nathan, MD, at Boston Children’s Hospital published the first paper showing one could use a virus to insert genes into blood-forming stem cells. In 2003, the ... Read More about After decades of evolution, gene therapy arrives
Research
How does the placenta protect unborn babies from COVID-19?
Evidence has shown that pregnancy is a risk factor for severe illness in women with COVID-19. A recent CDC study reviewed case reports of approximately 400,000 women aged 15 to 44 with symptomatic COVID-19. Those who were pregnant had a roughly tripled likelihood of ICU admission and invasive ventilation and 70 percent higher mortality. Yet ... Read More about How does the placenta protect unborn babies from COVID-19?
Patient Stories
A rebirth in Boston: Gene therapy turns 10
Lea la versión en español. Dec. 17 marks a decade since Agustín Cáceres was “renacido” — reborn. That’s how his parents, Alberto and Marcela, describe the day their son received his new gene. Born in 2010 with X-linked severe combined immunodeficiency (SCID-X1), Agustín spent the first few months of his life in isolation, at home ... Read More about A rebirth in Boston: Gene therapy turns 10
Clinical Care
In-hospital telehealth: Care innovation during the COVID-19 pandemic
The quick response, or QR, code — that square barcode symbol used by smartphone apps to link users with specific information — became an unexpected but essential tool for patient care at Boston Children’s Hospital in early spring. When the first patients with COVID-19 arrived, it quickly became clear that health care providers needed safer ... Read More about In-hospital telehealth: Care innovation during the COVID-19 pandemic
Our Community
Generations of excellence in surgical care: Dr. Emans and Dr. Hogue
Some surgeons follow their patients for years, even decades. This is true of Dr. John Emans, who has treated patients with complex spine conditions at Boston Children’s Hospital for more than 40 years. Beloved by patients, he is also widely respected by spine surgeons for his groundbreaking scoliosis research. Dr. Grant Hogue first met Dr. ... Read More about Generations of excellence in surgical care: Dr. Emans and Dr. Hogue
Clinical Care
Treating chronic pain virtually: A telehealth success story
When the COVID-19 pandemic forced shutdowns in March 2020, many clinicians found themselves faced with a whole new challenge: conducting much of patient care from their computers. Although telehealth was already a part of some physicians’ practices, others have had to learn how to adapt existing clinical skills to virtual platforms. But there have been ... Read More about Treating chronic pain virtually: A telehealth success story
Patient Stories
Devina’s story: Minimally invasive Foker process repairs esophageal atresia
Selina De Leon doesn’t have a background in medicine — but she does have experience being a mom. When the mother of four learned that her youngest child, Devina, had a rare birth defect, “I said, ‘esophageal what?’” she remembers. The condition, called esophageal atresia (EA), occurs when a baby’s esophagus develops in two separate ... Read More about Devina’s story: Minimally invasive Foker process repairs esophageal atresia
Clinical Care
At the forefront of kidney stone removal: Innovative approaches transform patient care
No longer considered just an adult problem, kidney stones increasingly affect children as well. The majority of children who cannot pass stones on their own can be treated with minimally invasive approaches such as extracorporeal shock wave lithotripsy, ureteroscopy, percutaneous nephrolithotomy, laparoscopic and robotic approaches and, rarely, open surgery. Now, two recent innovations provide additional ... Read More about At the forefront of kidney stone removal: Innovative approaches transform patient care
Patient Stories
Osteosarcoma patient gets chance to be a ‘normal college kid’
For almost half of his life, Michael Murray has had to grapple with cancer, including multiple relapses. One of his hardest setbacks was hearing that his cancer had returned just weeks before he was set to start his freshman year at Boston College. With the news, Michael worried that his future would be in jeopardy. ... Read More about Osteosarcoma patient gets chance to be a ‘normal college kid’