Blar i Bergen Open Research Archive på forfatter "Storli, Robert"
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Development of Small Molecules that Post-Transitionally Stabilizes the SMN Protein for the Treatment of Spinal Muscular Atrophy
Storli, Robert (Master thesis, 2019-08-31)Spinal Muscular Atrophy is an autosomal recessive neurodegenerative disorder, caused by the lack of SMN genes. Previous work done by Laboratory for Drug Discovery in Neurodegeneration (LDDN) at Brigham and Women’s Hospital ...