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In virtually all persons with amyotrophic lateral sclerosis (ALS) and in up to half of all cases of Alzheimer’s disease (AD) and frontotemporal dementia, a protein called TDP-43 is lost from its normal location in the nucleus of the cell. In turn, th
University of Virginia School of Medicine researchers have identified new insights and potential treatment approaches for muscle loss in myotonic...
UVA School of Medicine researchers have identified new insights and potential treatment approaches for muscle loss in myotonic dystrophy type 1 (DM1.
E-Mail Many cognitive neurodevelopmental disorders are a result of too many or too few copies of certain genes or chromosomes. To date, no treatment options exist for this class of disorders. MECP2 duplication syndrome (MDS) is one such disorder that primarily affects boys and results from a duplication spanning the methyl-CpG binding protein 2 (MECP2) locus located on the X chromosome. A preclinical study published from the laboratory of Dr. Huda Zoghbi, professor at Baylor College of Medicine and director of the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospit...