聚焦四大罕见病，逾三百位专家共促国际协作

Core Insights - The recent 2025 Complement Day Conference gathered nearly 300 top experts from over 10 countries to focus on rare diseases related to the complement system, including PNH, aHUS, gMG, and NMOSD [1][2] - The complement system plays a crucial role in innate immunity, but its dysfunction can lead to various inflammatory or autoimmune diseases, significantly impacting patient quality of life and survival [1] Group 1: Rare Diseases and Their Challenges - PNH, aHUS, gMG, and NMOSD are characterized by complex clinical presentations and high misdiagnosis rates due to insufficient awareness in primary healthcare settings [1] - These diseases progress rapidly and can affect multiple organ systems, leading to severe physical and economic burdens on patients and their families [1] Group 2: Advances in Diagnosis and Treatment - Recent advancements in diagnostic techniques and interdisciplinary collaboration have shifted the treatment of these rare diseases from traditional supportive care to precise targeted therapies [2] - The establishment of a national rare disease diagnosis and treatment network, comprising 419 hospitals, has created a quality control system at multiple levels, supported by a comprehensive case data registry of 1.74 million cases [2] Group 3: Policy and System Development - China has moved from a "policy vacuum" to a critical period of "system construction" in rare disease prevention and treatment, with significant achievements such as the inclusion of 207 rare diseases in the national directory [2] - The national medical insurance directory now covers over 90 rare disease drugs, with additional support measures like tax exemptions and expedited approvals for high-cost medications [2]