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Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2

Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial

A Randomized Clinical Trial to Evaluate the Effects of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2

Physical Activity, Motor Competence, Pulmonary Function, and Quality of Life in Children With Severe Spinal Disease

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

Longitudinal Study of Neurodegenerative Disorders

Coordination of Rare Diseases at Sanford

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