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Drug and Device Development

Old Drug, New Use
 Old Drug, New Use 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Nationwide Children's style illustration showing a row of houses with a prescription in front of it

Researchers have discovered an adult diabetes drug may provide a new and better way to treat children  with nephrotic syndrome.  Nephrotic syndrome is one of the most common kidney diseases seen in children. In most cases the cause is unknown. Nephrotic syndrome involves damage to the filtering units of the kidneys (glomeruli) leading to massive…

New Newborn Screening Approach Allows for Earlier Detection of Menkes Disease
 New Newborn Screening Approach Allows for Earlier Detection of Menkes Disease 1024 575 Natalie Wilson
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Targeted next generation DNA sequencing can enable improved clinical outcomes for any genetic disorder not detectable through other biochemical newborn screening methods. Menkes disease is a rare, X-linked recessive pediatric disease caused by gene mutations of the copper transporter gene, ATP7A, leading to low levels of copper in infants’ blood and brain, as well as abnormal…

Cannabidiol May Reduce Drug-Resistant Seizures by Half
 Cannabidiol May Reduce Drug-Resistant Seizures by Half 150 150 Brianne Moore
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Two studies demonstrate clinically significant response to oral liquid cannabidiol over placebo. Two recent studies – one published in The New England Journal of Medicine and one presented at the 2017 American Academy of Neurology annual meeting – indicate that a new plant-based, cannabis-derived treatment may be able to decrease the incidence of seizures in two complex…

Discovery to Drug Development: Expanding the Role of Academic Centers
 Discovery to Drug Development: Expanding the Role of Academic Centers 1024 575 Abbie Miller
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As more researchers at academic centers become involved in drug development, institutions are responding with support and guidance. Researchers at academic institutions regularly make discoveries about disease processes and potential therapeutic agents. Translational medicine is focused on moving these discoveries out of the laboratory and into the clinic where they can potentially help patients. But…