Penn Medicine researchers have developed a new targeted approach to prevent a toxicity seen in the sensory neurons of dorsal root ganglia after gene therapy to treat neurological disorders. It's an important hurdle to clear, ...
Nov 11, 2020
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In a new study, UC San Francisco scientists used maps of brain connections to predict how brain atrophy would spread in individual patients with frontotemporal dementia (FTD), adding to growing evidence that the loss of brain ...
Oct 14, 2019
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A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers.
Sep 19, 2019
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Muscle decline caused by ageing and certain diseases could be dramatically slowed by stopping a chain reaction that damages cells, new research shows.
Jun 7, 2019
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U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby's muscle control and kills nearly all of those with the most common type of the disease within a couple of years.
May 24, 2019
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A single misbehaving protein—called TDP-43—is behind 97 percent of amyotrophic lateral sclerosis (ALS) cases and 45 percent of frontotemporal dementia diagnoses. It also is found in 80 percent of chronic traumatic encephalopathy ...
Feb 27, 2019
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A new form of therapy may halt or even reverse a form of progressive vision loss that, until now, has inevitably led to blindness. This hyper-targeted approach offers hope to individuals living with spinocerebellar ataxia ...
Nov 1, 2018
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Groundbreaking research shows that neurological health depends as much on signals sent by the body's large, leg muscles to the brain as it does on directives from the brain to the muscles. Published today in Frontiers in ...
May 23, 2018
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Different Parkinson's-related brain disorders, called synucleionpathies, are characterized by misfolded proteins embedded in cells. Researchers in the Perelman School of Medicine at the University of Pennsylvania found that ...
May 9, 2018
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Spinal muscular atrophy (SMA) is a genetic disease that can leave infants with weak muscles and trouble breathing. Many with the disease die before age two. To help these patients, doctors need therapies that target the genetic ...
May 8, 2018
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