OHSU scientists have discovered a naturally occurring disease in monkeys that mimics a deadly childhood neurodegenerative disorder in people—a finding that holds promise for developing new gene therapies to treat Batten ...
Jul 27, 2018
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Researchers at Baylor College of Medicine, Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital and King's College London have discovered a treatment that improves the neurological symptoms in a ...
Feb 6, 2017
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A team led by researchers at Baylor College of Medicine has uncovered an unexpected mechanism that can explain a form of Batten disease called neuronal ceroid lipofuscinosis 8. The findings published in the journal Nature ...
Nov 5, 2018
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In a study of dogs, scientists showed that a new way to deliver replacement genes may be effective at slowing the development of childhood Batten disease, a rare and fatal neurological disorder. The key may be to inject viruses ...
Nov 11, 2015
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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 ...
Oct 10, 2019
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2017 is supposed to be the year that FDA finally approves a gene therapy. But last week, the company behind the first approved gene therapy in Europe, uniQure.com, announced that it won't "pursue the renewal of marketing ...
May 2, 2017
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Batten disease is a family of 13 rare, genetically distinct conditions. Collectively, they are the most prevalent cause of neurodegenerative disease in children, affecting 1 in 12,500 live births in the U.S. One of the Batten ...
Jun 30, 2020
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Researchers are working hard to fight childhood dementia and hope a new enzyme replacement therapy can help those unlucky enough to have such a debilitating disease.
Sep 1, 2017
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The New York Stem Cell Foundation (NYSCF) and Beyond Batten Disease Foundation (BBDF) today announce the availability of a unique and comprehensive collection of juvenile Batten (CLN3) disease induced pluripotent stem (iPS) ...
Oct 4, 2017
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Batten disease is a rare, fatal genetic disorder that affects children. Currently, no effective treatment exists for the disease, which ultimately kills all who are affected. Dachshunds also suffer from Batten disease, and ...
Feb 11, 2013
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