Medical Professional Publications

Groundbreaking Neuroscience Research and Clinical Innovations

(From the October 2015 Issue of PediatricsOnline)

From studies at the molecular level to the behavioral level, Neurosciences faculty members are investigating how neurological and neuromuscular disorders and conditions develop and working toward new treatment and prevention options. Here are summaries of several research and clinical developments from the past year. To receive future neurosciences announcements, be sure to subscribe to our e-newsletter PediatricsOnline.

SMA Clinical Trial Provides Hope for Families
Jerry Mendell, MD, and his team are conducting the world's first gene therapy clinical trial for SMA1 and are optimistic about preliminary results.
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Research Casts Doubt on Previously Accepted Genetic Cause of Juvenile Myoclonic Epilepsy
Specific mutations reported to cause JME are found to vary in frequency depending on ethnicity.
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Novel Technique Restores Gene and Function in Dysferlinopathy Animal Models
Clever cutting and delivery allow cell's own mechanisms to recombine needed DNA.
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Gene Therapy Trial Improves Walking Performance for Becker Muscular Dystrophy
In a Phase 1/2a trial, BMD patients receiving treatment showed improvements in walking ability over the placebo.
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Surprising Findings for Exon Skipping in Muscular Dystrophy
Which exons are skipped in therapy for DMD may determine whether significant improvement is made.
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Childhood Stroke Increases Adaptive and Social Behavior Impairment
After stroke, children who have normal cognition and behavior may still have impaired social adjustment and participation.
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