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  • In-office gene therapy for wet age-related macular degeneration is coming October 11, 2019
    Gene therapy is showing promise for one of the most common causes of blindness. Data presented today shows that six patients with wet age-related macular degeneration (AMD) have, so far, gone at least six months without the need for continued injections to control a disease that typically requires treatment every four to six weeks. Researchers […]
  • Powerful new genomics method can be used to reveal the causes of rare genetic diseases October 11, 2019
    A team led by a scientist at Scripps Research has invented a new genomics technique for tracking down the causes of rare genetic diseases.
  • Jumping genes can cause rare developmental disorders in children October 11, 2019
    The largest study of its kind into childhood developmental disorders has discovered that jumping genes cause genetic changes in some patients with undiagnosed neurodevelopmental diseases. The research from the Wellcome Sanger Institute and its collaborators in the NHS Regional Genetics services enabled genetic diagnoses for three children with previously undiagnosed rare developmental diseases who were […]
  • New customized drug treatment bypasses a single child's unique mutation within a year of diagnosis October 10, 2019
    An unprecedented case at Boston Children's Hospital shows that it's possible to do something that's never been done before: identify a patient's unique mutation, design a customized drug to bypass it, manufacture and test the drug, and obtain permission from the Food and Drug Administration (FDA) to begin treating the patient—all in less than one […]
  • Multigene testing for all breast cancer patients cost-effective October 10, 2019
    (HealthDay)—Unselected, multigene testing for all patients with breast cancer would be cost-effective in the United Kingdom and the United States, according to a study published online Oct. 3 in JAMA Oncology.
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