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Race for a Cure

A visit with Christian Lorson, Associate Professor of Veterinary Medicine

Published: - Topics: SMA Spinal_Muscular_Atrophy pediatric_neurodegenerative_disease infantile_death research
Topics: research clinical_trials

With research under his belt, Lorson hopes that his team can start to establish clinical trials to test the therapeutic programs they have developed. “The next step is to launch it into animal models and prove efficacy, showing that you can turn around the SMA phenotype [gene expression].”