Archive for research
Building a bladder-specific Botox
Botulinum toxins are bacterial neurotoxins that also have a range of therapeutic uses. In urology, injections of these potent substances can relax the bladder muscle, helping treat conditions such as overactive bladder and neurogenic bladder. Of the seven types of botulinum toxin, two have been approved by the U.S. Food and Drug Administration (FDA): botulinum ... Read More
Open-label placebo offers new treatment for disorders of gut-brain interaction in children
Pain-predominant disorders of gut-brain interaction (DGBIs) in children — such as functional abdominal pain (FAP) and irritable bowel syndrome (IBS) — can lead to disabling symptoms, poor quality of life, and high use of health care resources. While optimal treatment for these conditions remains elusive, we know that this patient population tends to have high ... Read More
Can intensive early behavioral therapy help children with Down syndrome?
“It’s like learning a new language. It’s a challenge, but a good challenge,” says Kristala of the new ways she has learned to connect with her 3-year-old son, Ellison, who has Down syndrome. Her favorite part of the Joint-Attention-Symbolic-Play-Engagement-Regulation (JASPER) clinical trial program at Boston Children’s Hospital came during the “Follow His Lead” week. “I ... Read More
Bringing genomics to community NICUs
About a year and a half ago, Robert Rothstein, MD, FAAP encountered a baby with a pattern of facial features and clinical findings that suggested a genetic syndrome. The available tests couldn’t pinpoint a diagnosis, and the family wanted a more definitive answer. So Rothstein and his colleagues transferred the newborn from Baystate Medical Center ... Read More
Surviving stem cell transplant: New hope when the donor isn’t a full match
To see Tara Daniels today, with a corporate job in marketing and about to close on a house, you’d never know what she’s been through, how thrilled she is to be alive. This month marks five years since she received a high-risk bone marrow transplant for a life-threatening blood cancer. Tara woke up feeling sick ... Read More
Addressing inequities in asthma by focusing on children’s environments
Asthma strikes children in low-income urban areas especially hard, more often sending them to the hospital. For more than 20 years, Dr. Wanda Phipatanakul at Boston Children’s Hospital has been investigating why — and seeking ways to level the playing field. “Children in low-income areas and children of color often aren’t diagnosed early enough with ... Read More
It takes a village: Creating best practices for personalized treatments
In 2019, doctors and scientists at Boston Children’s Hospital announced a medical landmark. In the space of one year, a team led by Timothy Yu, MD, PhD, pinpointed a one-of-a-kind genetic mutation in a girl named Mila with Batten disease, developed a custom drug called milasen to repair the mutation, and treated Mila with it. ... Read More
Solving neurodevelopmental mysteries, one gene, one child at a time
Suheil Day was born early, at 37 weeks. Aside from a slight head lag and mild muscle weakness, nothing seemed terribly amiss. But as the months progressed, he began having seizures. “At the age of 4 to 5 months, he started waking up screaming and crying excessively, his eyes rolling up into his head,” says ... Read More
With a dose of health equity, brachial plexus study enrolls more patients
What drives a parent to say yes or no to enrolling their child in research? When a surprisingly high percent of patient families said no to participating in a study of brachial plexus birth injury, orthopedic surgeon Andrea Bauer, MD, and her team decided to find out why. Along the way, they became versed in ... Read More
A promising new antiseizure drug tailored to newborns
Neonatal seizures can lead to serious consequences, including significant cognitive and motor disabilities, lifelong epilepsy, and death. They are often highly resistant to treatment, in part because seizures in newborns are fundamentally different from seizures in older children and adults. Yet they are treated in much the same way as older patients, with little change over the decades. Better treatment is ... Read More