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Risdiplam utilization, adherence, and associated health care costs for patients with spinal muscular atrophy: a United States retrospective claims database analysis.
Long-Term Risk of Stroke After Transcatheter Aortic Valve Replacement: Insights From the SwissTAVI Registry.
Advancing qualitative rare disease research methodology: a comparison of virtual and in-person focus group formats.
Neonatal screening and genotype-phenotype correlation of hyperphenylalaninemia in the Chinese population.
Vitamin D and skeletal health in autoimmune bullous skin diseases: a case control study.