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Summary of this study in pictogram: ABCA3 deficiency—variant-specific response to hydroxychloroquine. Step 1: systematically analyzed the clinical outcomes of patients with disease-causing ABCA3 variants treated with HCQ. Step 2: explored the effect of HCQ on 16 ABCA3 variants on the cellular level. Confocal images show E292V-variant-transfected A549 cells without and with exposure to HCQ (10 μM). Step 3: evaluated the relation of the HCQ response in vivo and in vitro.
