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Basket trials in rare diseases: a systematic review of current practices, methodological challenges, and future directions.
Lung volumetry of osteogenesis imperfecta type 3 subjects is not correlated with thoracic scoliosis and anthropometric data.
Coronary CT Anatomy-Based Prediction of Invasively Assessed Hemodynamic Significance in Middle-Aged Patients With Right Coronary Artery Anomaly: The NARCO Study.
Burden of illness and mortality in men with Adrenomyeloneuropathy: a retrospective cohort study.
A single-center observational study on long-term neurodevelopmental outcomes in children with tuberous sclerosis complex.