FDA rejects muscular dystrophy drug, says it doesn't work
The U.S. Food and Drug Administration has rejected an experimental drug for a common type of muscular dystrophy.
Oct 25, 2017
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The U.S. Food and Drug Administration has rejected an experimental drug for a common type of muscular dystrophy.
Oct 25, 2017
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Critical Path Institute (C-Path) and The Clinical Data Interchange Standards Consortium (CDISC) announce the open availability of a Duchenne Muscular Dystrophy Therapeutic Area User Guide ( TAUG-DMD v1.0), which describes ...
Oct 18, 2017
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A team of leading European clinicians and scientists presents a unique perspective on how to move forward in the development of exon skipping therapies to treat the severe muscle-wasting disease Duchenne Muscular Dystrophy ...
Oct 16, 2017
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Macrophages, a type of white blood cell involved in inflammation, readily take up a newly approved medication for Duchenne muscular dystrophy (DMD) and promote its sustained delivery to regenerating muscle fibers long after ...
Oct 16, 2017
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Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes ...
Oct 3, 2017
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New research published in The Journal of Physiology today suggests that enhancing breathing via the brain may limit deficiencies in respiratory capacity in Duchenne muscular dystrophy (DMD) patients.
Sep 27, 2017
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A new study published in the journal Nature Communications describes a breakthrough in research related to facioscapulohumeral muscular dystrophy (FSHD). The debilitating genetic disease - which has no approved treatment ...
Sep 15, 2017
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A mutated gene in patients lacking a nose has been identified by an international team, a first step toward understanding nose development and possible therapies for another condition.
Sep 15, 2017
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Researchers from the Perelman School of Medicine at the University of Pennsylvania have made a discovery about muscular dystrophy disorders that suggest new possibilities for treatment. In a study published today online in ...
Sep 7, 2017
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Mitochondria help injured muscle cells (myofibers) repair by soaking up calcium that enters from the site of injury and using it to trigger increased production of reactive oxygen species. Although reactive oxygen species ...
Sep 5, 2017
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