Intractable & Rare Diseases Research | The Imperative for National Legislation on Rare Diseases in China: A Policy Review and Call to Action

Intractable & Rare Diseases Research | From System Building to Global Engagement: Current Significance of and Future Directions for Rare Disease Research in China

Orphanet Journal of Rare Diseases | KLINSE Clinical Information Center Supports Rare Disease Diagnosis and Management

Intractable & Rare Diseases Research | A Multidisciplinary Management Cohort Study of Chinese Children with Fabry Disease

Intractable & Rare Diseases Research | Evaluating the Implementation Effectiveness of China's Rare Disease Policies Based on the PMC Index Model

Orphanet Journal of Rare Diseases | Exercise Intervention in Children and Young Adults with McArdle Disease: Feasibility, Acceptability, and Clinical Outcomes

Orphanet Journal of Rare Diseases | Pathogenic Mechanisms and Multimodal Therapies for Gorham-Stout Disease with Thoracic Involvement

Orphanet Journal of Rare Diseases | Real-World Efficacy of Glycerol Phenylbutyrate in Saudi Pediatric Patients with Urea Cycle Disorders