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Elevidys is the first-ever gene therapy for DMD, a neuromuscular disease characterized by progressive muscle weakness and atrophy that strikes primarily young boys

This actually makes the 505(b)(2) value-added path more attractive as it relies on clinical differentiation rather than just price

NH2-truncated human tau induces deregulated mitophagy in neurons by aberrant recruitment of Parkin and UCHL-1: implications in Alzheimers disease

Confirming the metabolic relevance of central GIPR signaling, neuronal loss of GIPR renders mice resistant to GIP-induced body weight loss and GIPR/GLP-1R co-agonism is more potent than selective GLP-1R agonism for weight loss 54