Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society | Sleep patterns and glycemia in adults with Cystic Fibrosis-Related Diabetes: a remote pilot study

Orphanet Journal of Rare Diseases | Oscillometry Reveals Expiratory Phase Lung Function Abnormalities in Patients with Late-Onset Pompe Disease

Orphanet Journal of Rare Diseases | Assessing Cardiometabolic Risk Using the Lipid- and BMI-Based SPISE Index in Pediatric Bardet-Biedl Syndrome

Orphanet Journal of Rare Diseases | Defining the Therapeutic Corridor of Stability in Enzyme Replacement Therapy for Pompe Disease

Orphanet Journal of Rare Diseases | Challenges and Opportunities in Rare Disease Management in the Middle East: A Multidimensional Analysis Based on Expert Consensus

Orphanet Journal of Rare Diseases | Evaluating Clinical Outcomes of HSCT versus ERT for Alpha-Mannosidosis

Orphanet Journal of Rare Diseases | A qualitative study on health-related quality of life in children and adolescents with hypohidrotic ectodermal dysplasia

Orphanet Journal of Rare Diseases | Predicting Hepatic Adenoma Risk in Glycogen Storage Disease Patients Using Biomarkers and Imaging Parameters