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The prevalence and long-term response to calcium channel blockers in patients with pulmonary arterial hypertension and positive vasoreactivity test - results of multicenter national registry (BNP-PL).
Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix.
🥇 Revisão sistemáticaLong-term effectiveness and safety of lomitapide in patients with homozygous familial hypercholesterolemia: an observational case series.
Current situation of osteogenesis imperfecta in Spain: results from a Delphi study.
Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.