As a new generation of gene therapy clinical trials shows promise to cure or halt the progression of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist-oncologist ...
May 26, 2016
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For the first time, researchers were able to demonstrate that children diagnosed with sickle cell disease showed improvement in lung function after treatment with hydroxyurea, a treatment that is underused despite its demonstrated ...
May 18, 2016
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Two-day-old Ellie Bailey squirms in a hospital bassinet and cries as her tiny left heel is squeezed and then pricked with a needle to draw a blood sample. An Indianapolis hospital technician quickly saturates six circles ...
May 13, 2016
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Gene therapies could one day offer life-saving treatments for diabetes, cancer, sickle cell disease and other illnesses that disproportionately affect ethnic minorities, yet many black Americans have profound reason to be ...
Mar 29, 2016
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The federal government is funding a monumental project to map 200,000 human genomes in an effort to understand the genetic causes of common diseases such as cardiovascular disease, diabetes, epilepsy and autism.
Jan 18, 2016
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Tiny vials of recently repaired blood cells are thriving in a Stanford incubator, proof that a powerful new gene-editing technique is fixing errant genes that cause so much human suffering.
Jan 12, 2016
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A study directed by Paul Frenette, M.D., is featured on the cover of today's print edition of Science. In experiments involving mice, Dr. Frenette and his colleagues have solved a mystery surrounding the development of hematopoietic ...
Jan 11, 2016
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A national sickle cell disease study involving Medical University of South Carolina researchers found that for some children with sickle cell disease, the drug hydroxyurea is as effective as blood transfusions to reduce blood ...
Jan 4, 2016
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An established drug for recurrent multiple myeloma might effectively be repurposed to improve the survival and day-to-day lives of patients with devastating sickle cell disease, according to revealing new research by a Feinstein ...
Dec 17, 2015
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A new study describes the development of pediatric pain measures for a National Institutes of Health Initiative aimed at helping doctors better evaluate and therefore better treat children who cope with chronic pain. Based ...
Dec 9, 2015
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