Blog | Page 16 of 159 | Boston Children's Answers

Gene therapy for adrenoleukodystrophy: Studies find both risks and benefits

Posted on October 16, 2024 by Nancy Fliesler | Clinical, Research

Tags: adrenoleukodystrophy, gene therapy, neurology

Cerebral adrenoleukodystrophy (CALD), portrayed in the film Lorenzo’s Oil, is a devastating disorder caused by a mutation on the X chromosome. Boys with CALD progressively lose their neurologic function and, if untreated, eventually become non-responsive. Most pass away within 10 years of diagnosis. Until recently, the only treatment for CALD was a blood (hematopoietic) stem ... Read More

Tagged: adrenoleukodystrophy, gene therapy, neurology

Catching up with Lucas eight years after his multivisceral transplant

Posted on October 15, 2024 by Veronica Giarla | Patient Stories

Tags: short bowel syndrome, transplant

Lucas and his mom, Heather, aren’t shy talking about his health journey and his multivisceral transplant. “If other families can learn something from Lucas’ story, it would be to keep advocating for your child and to trust that gut feeling,” Heather says. “Without organ donors, Lucas would not be here today.” Lucas was born with ... Read More

Tagged: short bowel syndrome, transplant

Emergency department or urgent clinic? Understanding your options if your child is injured

Posted on October 14, 2024 by Joanne Barker | Health and Parenting

Tags: emergency medicine, orthopedics, sports injury, sports medicine

Having an active or adventurous child often means comforting them when they come home with bumps or bruises. But when an injury is more serious, parents don’t always know where to seek the right level of care. The injury may look serious, but a trip to the emergency department may seem over the top — ... Read More

Tagged: emergency medicine, orthopedics, sports injury, sports medicine

Helping manage ‘sensitive issues’: How our psychosocial team helps kids with urologic concerns

Posted on October 14, 2024 by Jessica Cerretani | Our Community

Tags: urology

In Boston Children’s Department of Urology, urologists, pediatricians, and nurses aren’t the only ones who care for kids and their families. Psychologists, social workers, and child life specialists all work together to help children manage urologic conditions. We sat down with members of the department’s psychosocial team to learn more. Why did you choose to ... Read More

Tagged: urology

‘A lot better now’: Andrew’s recovery from Rocky Mountain spotted fever and a stroke

Posted on October 10, 2024 by Jessica Cerretani | Patient Stories

Tags: neuroimmunology, neuroinflammation, stroke

When Andrew was bitten by a tick in 2021, he and his parents could hardly have imagined that just a month later, he would be coping with a series of health problems that would culminate in a stroke. Andrew, then 7, didn’t even realize he’d been bitten until he began to feel sick with a ... Read More

Tagged: neuroimmunology, neuroinflammation, stroke

Shoring up heart muscle’s mini ‘managers’ to treat heart failure

Posted on October 8, 2024 by Nancy Fliesler | Basic/Translational, Research

Tags: arrhythmia, cardiac research, cardiology, cardiovascular genetics, gene therapy, heart, heart center

Our heart muscle is studded with tiny dyads, intricately designed structures that manage incoming electrical signals and calcium release to coordinate our heartbeats. Could gene therapy help maintain dyads’ structure and boost the function of failing hearts? A new study suggests it can. “We know that in heart failure from many causes, dyads become disorganized,” says ... Read More

Tagged: arrhythmia, cardiac research, cardiology, cardiovascular genetics, gene therapy, heart, heart center

EarlyBird: Addressing dyslexia through game play

Posted on October 1, 2024 by Nancy Fliesler | Primary Care, Therapeutics/Diagnostics/Devices

Tags: digital health, learning disabilities, primary care

Up to 10 percent of the population has dyslexia, yet many children are diagnosed only after struggling with reading for years. The stigma can cause low self-esteem, depression, and antisocial behavior. But imagine we could identify children at risk for dyslexia before they start formal reading instruction, then help them build the skills they need ... Read More

Tagged: digital health, learning disabilities, primary care

Will early intervention prevent asthma in school-age children?

Posted on September 30, 2024 by Nancy Fliesler | Clinical, Research

Tags: allergy, asthma, clinical trials, research

Asthma affects about 1 in 10 children, often sending them to the emergency room or causing them to miss school. Allergic conditions in young children — like food allergies or eczema — are often a precursor. Susceptible children begin producing IgE antibodies, which trigger allergic reactions and spur the “allergic march” toward asthma. IgE antibodies ... Read More

Tagged: allergy, asthma, clinical trials, research

Novel procedure may bridge the treatment gap for symptomatic flexible flatfoot

Posted on September 27, 2024 by Joanne Barker | Clinical, Research

Tags: flatfoot, lower extremity, orthopedics, surgery

Most children develop arches in their feet by early adolescence. About 20 percent, however, have pes planovalgus (PPV), also known as flexible flatfoot. Most children with PPV can participate in sports and other activities without issue, but others have ongoing pain in their feet that limit function and activities. Until recently in the U.S., children ... Read More

Tagged: flatfoot, lower extremity, orthopedics, surgery

Finding a possible genetic treatment for rare arrhythmias

Posted on September 18, 2024 by Albert McKeon | Basic/Translational, Research

Tags: arrhythmia, cardiac research, cardiology, cardiovascular genetics, congenital heart defect, heart, heart center, long qt syndrome, research

Variants in a gene that plays a key role in heart function can cause potentially life-threatening arrhythmia syndromes known as calmodulinopathy. Calmodulinopathy is rare and causes arrhythmias that are poorly treated by current options. Boston Children’s cardiologist William Pu, MD, believes he has found a promising custom genetic treatment: antisense oligonucleotides that deplete the disease-causing gene product. ... Read More

Tagged: arrhythmia, cardiac research, cardiology, cardiovascular genetics, congenital heart defect, heart, heart center, long qt syndrome, research