The conference will cover a range of topics including an introduction, patient registries, the clinical presentation of GABAAR disorders with a focus on necessary knowledge and outcome measures for clinical trials, the composition of GABAARs and specific information needed for treatment development, parent experiences on the importance of characterization, the critical understanding of variants and efficient screening methods, preclinical and off-label use of PBA for GABA-A variants, gene therapy for CNS disorders, and the current pipeline and strategies to attract industry, concluding with aroadmap to a cure.