Core Viewpoint - Duchenne Muscular Dystrophy (DMD) is transitioning from being untreatable to treatable due to the approval of innovative therapies, highlighting the importance of accessibility and affordability for patients in Suzhou [1][4] Group 1: Disease Overview - DMD is a severe hereditary neuromuscular disease that requires comprehensive care and support [4] Group 2: Proposed Systematic Approach - A closed-loop system for DMD management is suggested, encompassing screening, diagnosis, treatment, rehabilitation, and insurance [4] - Emphasis on deepening medical insurance reforms to explore separate payment options for rare disease medications and optimize specific policies to prevent patients from needing to be hospitalized for reimbursement [4] Group 3: Accessibility and Support - Recommendations include establishing a "green channel" for urgently needed drugs and incorporating them into universal insurance coverage to reduce out-of-pocket expenses [4] - The importance of prevention and rehabilitation as essential components of the healthcare system is highlighted, with suggestions to initiate newborn screening trials for early detection and intervention [4] Group 4: Collaborative Efforts - Suzhou is positioned to explore a comprehensive support model through multi-departmental collaboration, contributing positively to the "Healthy Suzhou" initiative [4]

Core Insights - The 2025 China Rare Disease Conference highlighted significant advancements in the diagnosis and treatment of rare diseases, showcasing a collaborative effort between medical professionals, researchers, and pharmaceutical companies [1][3][5] Healthcare System Developments - The national rare disease diagnosis and treatment collaboration network has expanded to 419 hospitals, with 626 hospitals participating in the rare disease reporting system [1][3] - The national medical insurance drug list now includes approximately 100 rare disease medications, covering 42 types of rare diseases, with 8.6 billion yuan allocated for rare disease drug payments in 2024, accounting for 7.7% of total drug payments during the agreement period [3][5] Research and Innovation - The conference emphasized the importance of research and innovation in rare disease treatment, with successful drug developments transforming previously untreatable conditions [5][6] - The establishment of the China Rare Disease Comprehensive Cloud Service Platform aims to enhance diagnostic applications and create a comprehensive map for rare disease treatment and support [5][8] Global Contributions and Collaboration - China is positioning itself as a key player in global rare disease governance, having established a rare disease reporting system covering 1.15 billion people and registering 1.64 million cases [8][9] - The government is actively promoting policies to accelerate the review and approval of rare disease medications and medical devices, while also establishing specialized funding and laboratories for rare diseases [8][9] Patient Care and Support - The conference highlighted various initiatives aimed at improving patient care, such as setting up rare disease outpatient clinics on the ground floor for easier access and implementing a lifelong follow-up system for patients [8][9] - The commitment to addressing the challenges faced by rare disease patients, including low awareness in grassroots areas and high drug costs, was underscored as a priority for ongoing efforts [9]