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- 2016
Novel treatments for rare rheumatologic disorders: analysis of the impact of 30？years of the US orphan drug actDOI: 10.1186/s13023-016-0443-x Keywords: Orphan drug act, Orphan drug development, Rare disease, Rare rheumatologic disease Abstract: Rare rheumatologic diseases are a heterogeneous group of conditions associated with high morbidity. As a whole group, rare rheumatologic diseases afflict millions of people demanding for effective therapies. Therefore, we analyzed the impact of the US Orphan Drug Act on the development of anti-rheumatic orphan drugs
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