MEDiscuss • Medical News • New avenue for developing treatments for genetic muscle-wasting disease
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New avenue for developing treatments for genetic muscle-wasting disease
Scientists have identified a promising new approach for developing drugs to treat Spinal muscular atrophy, the leading inherited cause of death in infants and toddlers. They have found that an enzyme called RhoA is overly active in a mouse model of this disease, and a common laboratory compound that blocks this enzyme can greatly increase survival.
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