Rand兰德：2025年评估针对突触核蛋白病的疾病改良疗法的累积、有意义的益处-会议记录和研究路线图（英文版）

DMTs might or might not change how patients experience symptoms over the short time frame of a clinical trial. If DMTs do slow disease progression, patients might not perceive the benefits in terms of delaying or preventing severe symptoms and disability. Understanding the value of DMTs for patients with synucleinopathies requires understanding the likely illness trajectory over the entire course of the disease, which can last many years. Therefore, it will be critical to develop frameworks a