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Translational studies allow us to learn from patients and to apply our discoveries in the lab to the clinic. Ongoing translational studies involving the Kolb Lab: VALIANT SMA Study – Prospective controlled trial of Valprioc Acid in Ambulant Adults with Spinal Muscular Atrophy. The Kolb Lab measures histone deacetylase activity and the expression levels of the SMN protein in patient blood samples. Small Heat Shock Protein Sequencing Project – We are undergoing a sequencing project of the small heat shock protein family in collaboration with The Molecular Pathology Laboratory at Ohio State University. Induced Pluripotent Stem Cell Project - In collaboration with the laboratory of Dr. Brian Kaspar, we are working to derive cell culture models of sporadic ALS. This project involves patient participation in the form of a single, small skin biopsy. Ongoing Clinical Trials in the Neuromuscular Division: Clinical Trial of Ceftriaxone in Subjects with ALS – Sponsor: NINDS/NIHA
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