Céline G. Saad, a Ph.D. candidate at Uniklinikum Heidelberg, presented the collaborative effort of the partners in CHARLIE at the APS Conference and in Kassel 08-10 March 2023. A poster of the primary result entitled "novel therapeutic strategies of glutaric aciduria type 1 (GA1) in a mouse model" was presented. Lively discussions took place about the news in this disease treatment strategies and the latest approaches that Charlie scientists are tackling in their research. The research group on neuropediatrics and metabolic medicine in Uniklinikum Heidelberg, headed by Prof. Dr. Kölker, is responsible for tackling new perspectives to find a suitable treatment strategy for rare disease patients. To understand human disorders pathology, they study on an animal model the disease development and potential new therapies strategies. The Heidelberg team is responsible to investigate the GA1 mouse model in different genetic backgrounds and to examine any sexual dimorphism between the animals. They characterize the Gcdh knockout mouse models on metabolic, molecular, and histological levels. Additionally, they analyze samples from different organs of these mice and compare them to matching controls. Further, by analyzing GA1 models with an additional Aass knockout, they study the therapeutic potential of this enzyme deficiency. All of this is possible through joining forces and expertise of this team with CHARLIE to support the Rare Disease Community both clinically and translationally.