Rare Disease Area Evaluation

Student: Mihaela Bet, 2023-2024

Amicus Therapeutics is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering high-quality medicines for people living with rare genetic diseases. Amicus is comprehensively investigating opportunities for a series of rare disorders through a multifaceted approach, including evaluating the market potential and assessing Amicus’ technological, research and development, and commercial capabilities to explore the feasibility of venturing into new therapeutic spaces. A crucial aspect of this endeavor involves engaging directly with physicians, patients, and research experts. This collaborative approach allows for invaluable insights into the unmet need and the potential of new therapies. The primary objective of this capstone project is to determine if an unmet demand for novel therapeutic solutions exists within a specific rare genetic disorder domain. With this approach, Amicus Therapeutics remains committed to advancing science and therapeutics for patients who show true unmet healthcare needs