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rare diseases

Sam Berns and Audrey Gordon, executive director of The Progeria Research Foundation and Berns's aunt, attend The New York Premiere Of HBO's "Life According To Sam" on October 8, 2013 in New York City. Thos Robinson/Getty Images for HBO hide caption

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Thos Robinson/Getty Images for HBO

FDA Approves First Drug For A Rapid Aging Disorder In Children

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A phlebotomist draws blood from a patient participating in a clinical trial for a cancer treatment. With hospitals focused on COVID-19, hundreds of studies are being put on hold. Jim West/Science Source hide caption

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Jim West/Science Source

Coronavirus Pandemic Brings Hundreds Of U.S. Clinical Trials To A Halt

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Nathaly Sweeney, a neonatologist at Rady Children's Hospital-San Diego and researcher with Rady Children's Institute for Genomic Medicine, attends to a young patient in the hospital's neonatal intensive care unit. Jenny Siegwart/Rady Children's Institute for Genomic Medicine hide caption

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Jenny Siegwart/Rady Children's Institute for Genomic Medicine

Fast DNA Sequencing Can Offer Diagnostic Clues When Newborns Need Intensive Care

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Alex, at 4 years and 11 months old, throws a toy football. Caroline Cheung-Yiu hide caption

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Caroline Cheung-Yiu

A Boy's Mysterious Illness Leads His Family On A Diagnostic Odyssey

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Zolgensma, a new drug approved by the FDA Friday, costs more than $2.1 million. It's made by AveXis, a drugmaker owned by pharmaceutical giant Novartis. Bloomberg/Bloomberg via Getty Images hide caption

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Bloomberg/Bloomberg via Getty Images

At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever

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Seven-year-old Carson Miller (left), and his brother, 5-year-old Chase Miller (right), both have a degenerative brain disease called MEPAN syndrome. There are only 13 people in the world who have it. Courtesy of Andrew Ross-Perry hide caption

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Courtesy of Andrew Ross-Perry

Medical Detectives: The Last Hope For Families Coping With Rare Diseases

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The Food and Drug Administration has seen a sharp increase in applications for drug to treat rare diseases. An oversight report found problems with how agency is handling them. Al Drago/CQ Roll Call/Getty Images hide caption

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Al Drago/CQ Roll Call/Getty Images

Connor Webb and his mother, Kim Webb, stand outside of their home in Huntington Beach, Calif. Connor was treated for a rare cancer at 16. He's well now but his mother is fighting for new cures in case the cancer comes back. Alex Welsh for NPR hide caption

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Alex Welsh for NPR