This segment focuses on the research, development, and commercialization of therapies for rare diseases, with a primary focus on central nervous system (CNS) disorders. The lead product, Fintepla (fenfluramine), is being evaluated in Phase III clinical trials for Dravet syndrome and Lennox-Gastaut syndrome, and in Phase II trials for other rare epileptic syndromes. Research and development activities include clinical trials, formulation development, and regulatory submissions. The company utilizes advanced technologies and methodologies in drug discovery, including small molecule development. The patient impact is significant, as these therapies address unmet medical needs in patients with debilitating conditions. Market positioning is enhanced by the focus on rare diseases, which often have less competition. Future opportunities include expanding the Fintepla label and developing additional therapies for rare neurological disorders. Regulatory and clinical aspects are critical, with a focus on FDA approval and post-market surveillance. Partnerships and collaborations, such as the one with Tevard Biosciences, are essential for advancing gene therapies for Dravet Syndrome and other genetic epilepsies.