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Platelet gene signatures detecting pulmonary artery stenosis in patients with pulmonary hypertension.
An open-label single-arm phase 1/2a study to evaluate the safety and exploratory efficacy of a VM202 in patients with Charcot-Marie-Tooth disease 1A.
Symptoms and correlates of depression and anxiety in children and adolescents with juvenile idiopathic arthritis.
Impact of COVID-19 infection in patients with inherited metabolic diseases: a National Multicenter Study from the French IMDs Healthcare Network for Rare Diseases.
Correlation of biochemical and imaging markers with hepatic adenoma in patients with glycogen storage disease: a retrospective single-center study.