Dargestellt sind die Veränderungen zum Vorjahr der Top-5 Medikamente im Bereich Orphan Drugs 2009. Der Umsatz für das Medikament Gilvec lag im Jahr 2009 um 13,8 Prozent höher als im Vorjahr.
Noonan and LEOPARD syndromes (NS and LS) belong to a group of related disorders called RASopathies characterized by abnormalities of multiple organs and systems including hypertrophic cardiomyopathy a
This statistic displays the number of accepted orphan designations per year in the United States from 2003 to 2023. In 2017, there were 477 orphan designations accepted, a record number so far. Orphan
Snyder Robinson Syndrome (SRS) is a rare disease associated with a defective spermine synthase gene and low intracellular spermine levels. In this study, a spermine replacement therapy was developed u
