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Rare disease stocks
We cover 46 companies grouped under the Rare disease theme. The median market value in the group is $6.02B. Across the group, the median move over the past trading day is −0.39%. 80% of the 44 with a 200-day average trade above it. Price change over the past year runs from −9.8% to +206.8% across the middle 80 percent of the group, with a median of +70.8%.
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Table
46 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return |
|---|---|---|---|---|
| VRTX | Vertex Pharmaceuticals Incorporated | $138.63B | Large cap | +40.66% |
| ALNY | Alnylam Pharmaceuticals, Inc. | $32.12B | Large cap | −47.38% |
| INSM | Insmed Incorporated | $27.16B | Large cap | −7.54% |
| ROIV | Roivant Sciences Ltd. | $26.55B | Large cap | +206.51% |
| JAZZ | Jazz Pharmaceuticals plc | $16.89B | Large cap | +100.35% |
| ASND | Ascendis Pharma A/S | $16.41B | Large cap | +29.66% |
| BBIO | BridgeBio Pharma, Inc. | $15.52B | Large cap | +60.05% |
| CORT | Corcept Therapeutics Incorporated | $12.95B | Large cap | +71.81% |
| BMRN | BioMarin Pharmaceutical Inc. | $12.70B | Large cap | +11.85% |
| ARWR | Arrowhead Pharmaceuticals, Inc. | $12.52B | Large cap | +308.99% |
| CYTK | Cytokinetics, Incorporated | $11.01B | Large cap | +109.36% |
| IONS | Ionis Pharmaceuticals, Inc. | $10.56B | Large cap | +48.21% |
| KYMR | Kymera Therapeutics, Inc. | $10.53B | Large cap | +197.09% |
| PTGX | Protagonist Therapeutics, Inc. | $9.80B | Mid cap | +146.62% |
| IMVT | Immunovant, Inc. | $9.07B | Mid cap | +189.13% |
| RYTM | Rhythm Pharmaceuticals, Inc. | $7.78B | Mid cap | +9.35% |
| SRRK | Scholar Rock Holding Corporation | $7.31B | Mid cap | +74.18% |
| ORKA | Oruka Therapeutics, Inc. | $6.58B | Mid cap | +555.07% |
| LQDA | Liquidia Corporation | $6.31B | Mid cap | +154.86% |
| TVTX | Travere Therapeutics, Inc. | $6.24B | Mid cap | +284.86% |
| DNTH | Dianthus Therapeutics, Inc. | $6.13B | Mid cap | +356.93% |
| PTCT | PTC Therapeutics, Inc. Common Stock | $6.07B | Mid cap | +46.02% |
| KNSA | Kiniksa Pharmaceuticals International, plc | $6.05B | Mid cap | +133.47% |
| MIRM | Mirum Pharmaceuticals, Inc. | $5.98B | Mid cap | +31.96% |
| GRFS | Grifols, S.A. | $5.38B | Mid cap | −19.39% |
| DYN | Dyne Therapeutics, Inc. | $4.92B | Mid cap | +93.26% |
| EWTX | Edgewise Therapeutics, Inc. | $4.80B | Mid cap | +207.13% |
| MANE | Veradermics, Incorporated | $4.29B | Mid cap | +172.32% |
| CLDX | Celldex Therapeutics, Inc. | $3.19B | Mid cap | +79.51% |
| IRON | Disc Medicine, Inc. | $3.17B | Mid cap | +37.90% |
| ARQT | Arcutis Biotherapeutics, Inc. | $3.12B | Mid cap | +60.64% |
| VRDN | Viridian Therapeutics, Inc. | $2.75B | Mid cap | +30.71% |
| VERA | Vera Therapeutics, Inc. | $2.65B | Mid cap | +67.21% |
| RARE | Ultragenyx Pharmaceutical Inc. | $2.61B | Mid cap | −12.10% |
| PHVS | Pharvaris N.V. | $2.60B | Mid cap | +55.59% |
| TRVI | Trevi Therapeutics, Inc. | $2.55B | Mid cap | +124.00% |
| NKTR | Nektar Therapeutics | $2.52B | Mid cap | +151.95% |
| BCRX | BioCryst Pharmaceuticals, Inc. | $2.51B | Mid cap | +15.38% |
| COAG | Hemab Therapeutics Holdings, Inc. | $2.30B | Mid cap | +44.68% |
| MLYS | Mineralys Therapeutics, Inc. | $2.28B | Mid cap | +70.22% |
| PVLA | Palvella Therapeutics, Inc. | $2.22B | Mid cap | +173.39% |
| AUPH | Aurinia Pharmaceuticals Inc. | $2.20B | Mid cap | +36.58% |
| ADMA | ADMA Biologics, Inc. | $2.16B | Mid cap | −44.17% |
| ZBIO | Zenas BioPharma, Inc. | $2.15B | Mid cap | +97.73% |
| STOK | Stoke Therapeutics, Inc. | $2.14B | Mid cap | +71.45% |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $2.03B | Mid cap | −13.45% |
Covers ~22,000 companies across the US, Europe and Asia-Pacific (ex mainland China, Hong Kong and India), refreshed nightly. Every listed stock opens as a full analysis for $9.90.