DMPQ is a potent inhibitor of human platelet-derived growth factor receptor β (PDGFRβ, IC50 = 80 nM). It displays greater than 100-fold selectivity for PDGFRβ over other tyrosine and serine/threonine kinases. DMPQ effectively blocks the PDGF-mediated increase in the protein, Survival Motor Neuron, in fibroblasts from patients with spinal muscular atrophy.
