中国罕见病高峰论坛召开，多方专家共探罕见病事业发展之路

Core Viewpoint - Rare diseases are a global public health issue and should be prioritized in health investment strategies to address the challenges they pose [1][2]. Group 1: Importance of Rare Diseases - The rare disease population is among the most vulnerable and requires prioritization to avoid falling behind in healthcare [2]. - There is a global consensus that rare diseases are not only medical challenges but also involve social, economic, and human rights issues [2]. Group 2: Current Status in China - China has defined 207 rare diseases in its catalog, but this is limited compared to over 10,000 rare diseases globally, indicating a significant lag [2]. - Legislative measures are urgently needed to systematically advance the prevention and treatment of rare diseases in China [2]. Group 3: Innovation and Development - Comprehensive support for the development of the rare disease industry is essential, including promoting innovative technologies and enhancing international competitiveness [3]. - China has shown significant strength in innovative drug development for rare diseases, moving from a follower to a key player in the field [4][6]. Group 4: Future Prospects - The treatment of rare diseases is shifting from symptomatic treatment to addressing root causes, with gene therapy and editing technologies providing new possibilities [5]. - China is expected to continue narrowing the gap with Western countries and may achieve breakthroughs in more areas of rare disease treatment [6]. Group 5: Collaborative Efforts - The China Rare Disease Summit serves as a major academic event, facilitating discussions among experts from various fields to promote the development of rare disease initiatives in China [6].