Sulfonylurea in the treatment of neonatal diabetes mellitus children with heterogeneous genetic backgrounds

Abstract The pathogenic base of neonatal diabetes mellitus (NDM) is highly heterogeneous. Sulfonylurea (SU) has been successfully applied in majority of NDM patients with K We identified 16 patients with NDM. These patients underwent SU titration and were followed after successful SU monotherapy. All patients were sequenced for all exons and adjacent intron-exon junctions of Of the 16 patients, 15 (94%) reached glycemic goal (7–10 mmol/L) after SU monotherapy except one patient with the Our study showed that SU monotherapy resulted in satisfactory glycemic control in most of the patients with NDM whose genetic defects are heterogeneous. The usage of SU may be considered as first-line therapy for patients with NDM in developing countries where effective genetic screening is not established.