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Tough cookie: Steroid therapy helps Alessandra thrive with Diamond-Blackfan anemia

Posted on by | Patient Stories
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Two-year-old Alessandra is many things. She’s sweet, happy, curious, and, according to her parents, Ralph and Irma, a budding food critic. “She’s a food snob!” Ralph says, laughing. “She recognizes the expensive pasta, the homemade versus store-bought food.” But above all, Alessandra is resilient, and her parents have known that from the very start: At ... Read More
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The exciting life of Jack, the first successful fetal cardiac intervention patient

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Jack Miller sometimes reaches out to his cardiologist, Dr. Wayne Tworetzky, with updates on his life.  Recently, their conversations centered around Jack pushing himself while training to be a police officer. The physical endurance Jack needed for training was another example of how the fetal cardiac intervention that Dr. Tworetzky and the specialists of the Fetal Cardiology Program performed on ... Read More
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Mark’s winning pass with cochlear implants

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Mark Bradshaw wanted to break out of his parents’ protective shell — as many teens do when they start pushing for more independence. Mark’s parents, however, hesitated. The issue? Mark, who is 15 and has had cochlear implants since early childhood, wanted to play tackle football. Access to sound and language Mark was diagnosed with ... Read More
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A new druggable cancer target: RNA-binding proteins on the cell surface

Posted on by | Research, Therapeutics/Diagnostics/Devices
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In 2021, research led by Ryan Flynn, MD, PhD, and his mentor, Nobel laureate Carolyn Bertozzi, PhD, opened a new chapter in biology, characterizing a new kind of player on the cell surface: glycoRNAs. Extending this discovery recently in Cell, Flynn and colleagues showed that glycoRNAs form highly organized clusters with RNA-binding proteins on the ... Read More
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Jackie’s dreams of playing professional soccer back on track after ACL surgery

Posted on by | Patient Stories
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From her dorm in Newcastle, England, Jackie Zapata can hear fans roaring in the soccer stadium a few blocks away. Soccer is a major pastime in her adopted home. “The city gets amped up anytime there’s a game,” she says. “It’s a huge part of the culture here.” This enthusiasm makes Newcastle a good place ... Read More
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Understanding and treating Mason’s congenital nevus

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Kim and Ryan noticed their son’s birthmark almost immediately after he was born. They knew birthmarks were common, but Mason’s was different — it covered a large portion of his right leg and calf. Their doctor classified it as a giant congenital melanocytic nevus and suggested they monitor it closely, but that didn’t answer all ... Read More
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Unique data revealed just when Mickey’s heart doctors could operate

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When Mikolaj “Mickey” Karski’s family traveled from Poland to Boston to get him heart care, they weren’t thinking about pressure-volume (PV) loops. His parents simply wanted him to receive treatment for a complex condition that he couldn’t get back home. Little did they know the mathematical computational power of those PV loops would play a ... Read More
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No limitations: How Flora found answers for MOG antibody disease

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Flora Ringler’s fifth birthday didn’t turn out as she had hoped. She and her family were vacationing in New Hampshire last year when she started feeling tired. But she wasn’t just worn out from celebrating: She also developed a bad headache and started throwing up. Concerned, her mom, Maureen, contacted a pediatrician, who thought she ... Read More
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Forecasting the future for childhood cancer survivors

Posted on by | Clinical, Research
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Children are much more likely to survive cancer today than 50 years ago. Unfortunately, as adults, many of them develop cardiovascular disease, secondary cancers, or both, believed to result from the toxic effects of chemotherapy and radiation. But the full risk picture for survivors hasn’t been clear. “Patients want to know how long they’ll live ... Read More
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Genetic causes of congenital diarrhea and enteropathy come into focus

Posted on by | Clinical, Research
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Congenital diarrheas and enteropathies are rare and devastating for infants and children. Treatments have consisted mainly of fluid and nutritional management. But in recent years, targeted dietary and drug therapies have emerged based on genetic discoveries. Now, research led by Boston Children’s Hospital, The Hospital for Sick Children in Toronto, and UCLA takes a major ... Read More
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