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| VRTXVRTXVertex Pharmaceuticals Incorporated | Vertex Pharmaceuticals Incorporated | $523.93 | $132.8B |
| ALNYALNYAlnylam Pharmaceuticals, Inc. | Alnylam Pharmaceuticals, Inc. | $224.03 | $30.0B |
| INSMINSMInsmed Incorporated | Insmed Incorporated | $93.39 | $20.4B |
| ASNDASNDAscendis Pharma A/S | Ascendis Pharma A/S | $243.37 | $15.2B |
| BBIOBBIOBridgeBio Pharma, Inc. | BridgeBio Pharma, Inc. | $68.56 | $13.4B |
| IONSIONSIonis Pharmaceuticals, Inc. | Ionis Pharmaceuticals, Inc. | $42.93 | $7.1B |
| PTCTPTCTPTC Therapeutics, Inc. | PTC Therapeutics, Inc. | $60.59 | $5.1B |
| TVTXTVTXTravere Therapeutics, Inc. | Travere Therapeutics, Inc. | $50.08 | $4.7B |
| RARERAREUltragenyx Pharmaceutical Inc. | Ultragenyx Pharmaceutical Inc. | $15.65 | $1.5B |
| PHARPHARPharming Group N.V. | Pharming Group N.V. | $11.20 | $769.0M |