The Miami Project to Cure Paralysis, a Center of Excellence at the University of Miami Miller School of Medicine, in collaboration with Scholar Rock, a biotechnology company focused on discovering and developing drugs that ...
Jul 10, 2017
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A major step forward has been made by researchers at the University of St Andrews towards finding new treatments for Spinal Muscular Atrophy (SMA), the commonest genetic cause of infant death.
Apr 18, 2018
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(Medical Xpress)—Researchers from Plymouth University Peninsula Schools of Medicine and Dentistry have been part of an international team led by the University of Edinburgh, who have identified that a chemical found in ...
Mar 4, 2014
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Scientists at the University of Malta and the Institut de Génétique Moléculaire de Montpellier (CNRS/Université de Montpellier) have shown that fruit flies and brewer's yeast can reveal clues about the cause of Spinal ...
Jun 29, 2015
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In a new publication that appears in Human Molecular Genetics, the laboratory of Christine DiDonato, PhD reports on their pharmacological characterization of the drug RG3039, demonstrating that it can extend survival and ...
Jun 4, 2013
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Research from the University of Warwick indicates that most people are in favour of newborn screening for the potentially deadly condition spinal muscular atrophy (SMA). The study Newborn genetic screening for spinal muscular ...
Dec 5, 2017
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In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene abeparvovec) alone or ...
Jun 25, 2024
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Spinal muscular atrophy (SMA) is a severe neurological disease for which there is presently no cure, although current therapies can alleviate symptoms. In the search for better treatment options, scientists at DZNE and the ...
8 hours ago
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