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Alnylam receives positive opinion for orphan drug designation in the European Union for ALN-TTRsc


Wednesday, 2 Apr 2014 08:00am EDT 

Alnylam Pharmaceuticals Inc:Says that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending ALN-TTRsc for designation as an orphan medicinal product for the treatment of transthyretin (TTR)-mediated amyloidosis.ALN-TTRsc is currently in a pilot Phase 2 clinical trial for the treatment of ATTR patients with TTR cardiac amyloidosis; this study is aimed at evaluating the tolerability of ALN-TTRsc in about 15 patients. 

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