Intractable & Rare Diseases Research | Progress in the Research and Pharmacoeconomic Evaluation of Drugs and Devices for Rare Diseases in China

Orphanet Journal of Rare Diseases | Nutritional Assessment and Health Risks of Special Low-Protein Foods: A Narrative Review

Orphanet Journal of Rare Diseases | Recessive Congenital Methemoglobinemia: A Systematic Review and Genotype–Phenotype Correlation Analysis

Orphanet Journal of Rare Diseases | Intranasal Oxytocin in Infancy Improves Swallowing Function and Disease Trajectory in Prader-Willi Syndrome

Orphanet Journal of Rare Diseases | A Systematic Review of Clinical Features of Infantile Epileptic Spasms Syndrome

Orphanet Journal of Rare Diseases | Pathogenic Mechanisms of ATP1A3 Mutation-Related Alternating Hemiplegia of Childhood and Bipolar Disorder, and the Therapeutic Potential of Lithium

Orphanet Journal of Rare Diseases | Personalized Home-Based Exercise Training Improves Aerobic Capacity and Quality of Life in Children with Marfan and Loeys-Dietz Syndromes

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