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Orphan Drug Approvals in 2014

In Regulation & Government by Cameron

[tab_nav type=”two-up”][tab_nav_item title=”FDA Orphan Drug Approvals” active=”true”][tab_nav_item title=”EMA Positive Opinions for Orphan Drugs” active=””][/tab_nav][tabs][tab active=”true”] [accordion] [accordion_item title=”Trametinib – melanoma”] Generic Name: trametinib and dabrafenib …

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New Rare Syndromes: June-July

In Reports by Cecile

Novel form of congenital lipodystrophy and fatty liver disease due to biallellic loss-of-function mutations in PCYT1A in two unrelated patients The authors identified two unrelated …