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Rare & Orphan Disease

Drug developers focused on rare and orphan indications.

5 Tickersbiotech
ALNYALNYLAM PHARMACEUTICALS, INC.PharmaNASDAQBMRNBIOMARIN PHARMACEUTICAL INCPharmaNASDAQIONSIONIS PHARMACEUTICALS INCPharmaNASDAQSRPTSarepta Therapeutics, Inc.PharmaNASDAQVRTXVERTEX PHARMACEUTICALS INC / MAPharmaNASDAQ

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