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Sickle cell gene therapy and boosting fetal hemoglobin: A 75-year history

Posted on by | Clinical Care, Research
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Ed. Note: This post updates an earlier post from 2018. In a landmark decision today, the Food and Drug Administration (FDA) approved two gene therapies for sickle cell disease. One of them, Casgevy, has deep scientific roots at Boston Children’s Hospital — and is also the first therapy using CRISPR gene editing to gain FDA ... Read More
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An off-the-shelf tamponade kit provides surgeons with ‘the luxury of time’ during a life-threatening emergency

Posted on by | Clinical Care
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It was a late Friday afternoon in April when the call came: A young boy was being transferred to Boston Children’s emergency department after swallowing a button battery. Although his mother had previously taken him to another local hospital when she noticed he wasn’t eating or drinking much, the battery ingestion had been missed until ... Read More
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Writing the book on infantile spasms: Charlotte’s story

Posted on by | Patient Stories
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When Charlotte, 3, developed infantile spasms last year, her parents, Kate and Brett, entered a world they didn’t know how to navigate. Inspired by their daughter and the care she received at the Epilepsy Center at Boston Children’s Hospital, they set out to help other families facing similar experiences. Nervous parent instincts prove right “I ... Read More
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Could ADHD be diagnosed genetically?

Posted on by | Clinical, Research
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Despite it being very common, ADHD is often missed until a child reaches age 7 or older. By that time, they have likely been struggling socially and in school. Could early genetic testing be part of the solution? Anne Arnett, PhD, in partnership with Ryan Doan, PhD, at Boston Children’s Hospital, set out to see ... Read More
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How hearing aids opened up a world of sound for Harry

Posted on by | Patient Stories
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Maybe it’s unusual to bring a musical instrument to an audiology appointment, but that didn’t stop Harry King. When he picked up his new hearing aids from Boston Children’s Hospital, he brought his ukulele. After a procedure to drain fluid from his middle ears the year before, Harry had become obsessed with sounds of all ... Read More
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All in the family: One family’s shared link to epilepsy and fight for answers

Posted on by | Patient Stories
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One of the wonders of parenthood is seeing the traits you pass along to your kids, like your eyes or your laugh. For the Anderson family, however, one shared trait is more complex: a mutation in the NPRL3 gene that leads to epilepsy. It took four generations to uncover the family’s genetic link to the ... Read More
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Lessons from Emma: Mom shares how RNS has changed their lives

Posted on by | Patient Stories
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Emma’s journey with the Epilepsy Center at Boston Children’s Hospital began three years ago when she came to Boston Children’s with her parents for help managing her seizures. In Boston, Emma, Lynda, and Danny found answers to their questions, a (temporary) new home, and hope for Emma’s future as she embarked on responsive neurostimulation (RNS) ... Read More
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From ECMO to an active childhood: Nolan’s journey with congenital diaphragmatic hernia

Posted on by | Patient Stories
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Nolan Griffin knows how to use a stethoscope and rarely enters Boston Children’s Hospital without his medical kit. But Nolan isn’t a doctor ­— at least not yet. For now, the busy 2-year-old is a patient in the hospital’s Congenital Diaphragmatic Hernia Center, where he’s been receiving care since before he was born. Although Nolan’s ... Read More
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Timing is everything: How circadian rhythms influence our brains

Posted on by | Basic/Translational, Research
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Why are we mentally sharper at certain times of day? A study led by Jonathan Lipton MD, PhD, at Boston Children’s Hospital spells out the relationship between circadian rhythms — the body’s natural day/night cycles — and the brain connections known as synapses. The work is the first to provide a cellular and molecular explanation ... Read More
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What you should know about hemangiomas, according to a pediatric dermatologist

Posted on by | Health and Parenting
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When parents discover that their infant has a hemangioma, a lot of questions usually follow. To find answers for the most common questions and share some important facts about the condition, we spoke with Dr. Marilyn Liang, co-director of the Vascular Anomalies Center (VAC) and an attending physician in the Dermatology Program at Boston Children’s ... Read More
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