Small-scale trials with significant demands.

In the area of rare disease research, the unmet needs of patients have become a driving force behind the development of innovative scientific breakthroughs. Due to the limited and hard-to-find patient groups associated with rare diseases, conducting studies in this field presents significant challenges. Each study necessitates a highly proficient team capable of navigating complexities, addressing uncertainties, and overcoming obstacles that are unique to rare diseases. This level of proficiency is crucial for introducing novel therapies to patients and their families, offering hope and potential life-changing treatments to those who are often overlooked in traditional medical research.