Treatment Options for Patients with Maturity-Onset Diabetes of the Young (MODY): A Systematic Review of Literature: 2026 Update
Abstract
Introduction: Maturity-onset diabetes of the young (MODY) is a rare monogenic form of diabetes characterized by impaired insulin secretion and genetic and clinical heterogeneity. Accurate molecular diagnosis enables precision medicine by guiding gene-specific treatment, improving glycemic control, and avoiding unnecessary therapies. Since our previous systematic review in 2020, new studies have further clarified therapeutic strategies and emerging treatment options. This review updates the evidence on pharmacological management of MODY and its clinical implications.
Methods: A systematic review was conducted following PRISMA 2020 recommendations. PubMed was searched for studies published between April 2020 and October 2025. Eligible studies included individuals with genetically confirmed MODY receiving pharmacological treatment, therapeutic switches, or adjunctive therapies. Data were extracted and synthesized qualitatively due to heterogeneity in study design and outcome reporting.
Results: Ninety-one studies were included. Strong evidence confirms sulfonylureas (SUs) as first-line therapy in Hepatocyte Nuclear Factor 1A (HNF1A)-, Hepatocyte Nuclear Factor 4A (HNF4A)-, ATP-binding cassette transporter sub-family C member 8 (ABCC8)-, and potassium inwardly rectifying channel subfamily J member 11 (KCNJ11)-MODY, frequently enabling improved glycemic control and transition from insulin. Glucokinase (GCK)-MODY remains managed without pharmacological treatment outside pregnancy,…
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