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| ALNYALNYAlnylam Pharmaceuticals, Inc. | Alnylam Pharmaceuticals, Inc. | $227.28 | $30.4B |
| ASNDASNDAscendis Pharma A/S | Ascendis Pharma A/S | $242.99 | $15.2B |
| BMRNBMRNBioMarin Pharmaceutical Inc. | BioMarin Pharmaceutical Inc. | $56.63 | $11.0B |
| IONSIONSIonis Pharmaceuticals, Inc. | Ionis Pharmaceuticals, Inc. | $43.91 | $7.3B |
| PTCTPTCTPTC Therapeutics, Inc. | PTC Therapeutics, Inc. | $63.04 | $5.3B |
| SRRKSRRKScholar Rock Holding Corporation | Scholar Rock Holding Corporation | $41.96 | $5.1B |
| TVTXTVTXTravere Therapeutics, Inc. | Travere Therapeutics, Inc. | $52.43 | $4.9B |
| DNLIDNLIDenali Therapeutics Inc. | Denali Therapeutics Inc. | $18.66 | $3.0B |
| MAZEMAZEMaze Therapeutics, Inc. | Maze Therapeutics, Inc. | $31.95 | $1.8B |
| RARERAREUltragenyx Pharmaceutical Inc. | Ultragenyx Pharmaceutical Inc. | $15.92 | $1.6B |