A sickle cell first: Base editing, a new form of gene therapy, leaves Branden feeling ‘more than fine’
Though he doesn’t remember it, Branden Baptiste had his first sickle cell crisis at age 2. Through elementary school, he was in and out of the hospital with pain episodes, not knowing why. As he got older, he learned he had sickle cell disease. His red blood cells were forming sickle shapes and getting stuck ... Read More about A sickle cell first: Base editing, a new form of gene therapy, leaves Branden feeling ‘more than fine’
Fuel to be faster: Studying the effects of low energy availability at the Boston Marathon
Like many sports medicine specialists, Kristin Whitney, MD, MA, suspected that many of the issues she treats in runners — bone stress injuries, anemia, decreased response to training, and reduced endurance to name a few — stemmed from insufficient nutrition. Key takeaways Runners with indicators of low energy availability had slower finish times and more ... Read More about Fuel to be faster: Studying the effects of low energy availability at the Boston Marathon
Making pediatric health equity research truly equitable: An EDI review process
A burgeoning number of studies are examining pediatric health equity, diversity, and inclusion (EDI). But if not done right, health equity research can do a disservice, perpetuating biases and wrong assumptions that actually exacerbate inequities. To guide EDI-related studies, the Institutional Review Board (IRB) at Boston Children’s Hospital (through Tina Young Poussaint, MD, and Susan ... Read More about Making pediatric health equity research truly equitable: An EDI review process
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 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 about Gene therapy for adrenoleukodystrophy: Studies find both risks and benefits
Will early intervention prevent asthma in school-age children?
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 about Will early intervention prevent asthma in school-age children?
Novel procedure may bridge the treatment gap for symptomatic flexible flatfoot
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 about Novel procedure may bridge the treatment gap for symptomatic flexible flatfoot