Free tool
Rare disease stocks
We cover 68 companies grouped under the Rare disease theme. The median market value in the group is $6.86B. Across the group, the median move over the past trading day is +1.08%. 78% of the 65 with a 200-day average trade above it. Price change over the past year runs from −13.8% to +210.4% across the middle 80 percent of the group, with a median of +51.3%.
Other lists in this group
SaaSAI applicationsCybersecuritySemiconductor designSemiconductor equipmentFintech softwarePayment processingEcommerce marketplacesStreaming mediaGaming softwareOncologyRare diseaseMedical devicesCell therapyDiagnosticsRegional banksAsset managementGold miningCryptocurrency and digital assetsSpecialty chemicalsAll stocks
Table
68 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return |
|---|---|---|---|---|
| VX1.DE | Vertex Pharmaceuticals Incorporated | $140.77B | Large cap | +40.14% |
| VRTX34.SA | Vertex Pharmaceuticals Incorporated | $140.14B | Large cap | −83.17% |
| VRTX | Vertex Pharmaceuticals Incorporated | $140.04B | Large cap | +42.14% |
| 0QZU.L | Vertex Pharmaceuticals Incorporated | $112.52B | Large cap | +42.39% |
| ARGX | argenx SE | $63.78B | Large cap | +50.52% |
| A1RG34.SA | argenx SE | $63.17B | Large cap | +44.49% |
| 0HD2.L | Alnylam Pharmaceuticals, Inc. | $35.05B | Large cap | −46.85% |
| ALNY | Alnylam Pharmaceuticals, Inc. | $32.12B | Large cap | −45.88% |
| DUL.DE | Alnylam Pharmaceuticals, Inc. | $31.87B | Large cap | −11.75% |
| A1LN34.SA | Alnylam Pharmaceuticals, Inc. | $31.78B | Large cap | −49.89% |
| INSM | Insmed Incorporated | $27.07B | Large cap | −3.45% |
| ROIV | Roivant Sciences Ltd. | $26.26B | Large cap | +213.32% |
| JAZZ | Jazz Pharmaceuticals plc | $16.89B | Large cap | +108.87% |
| ASND | Ascendis Pharma A/S | $16.59B | Large cap | +32.89% |
| BBIO | BridgeBio Pharma, Inc. | $15.85B | Large cap | +70.01% |
| CORT | Corcept Therapeutics Incorporated | $13.36B | Large cap | +79.56% |
| 0JAV.L | Insmed Incorporated | $12.89B | Large cap | −6.25% |
| BMRN | BioMarin Pharmaceutical Inc. | $12.80B | Large cap | +13.03% |
| REC.MI | Recordati Industria Chimica e Farmaceutica S.p.A. | $12.60B | Large cap | +2.09% |
| B1MR34.SA | BioMarin Pharmaceutical Inc. | $12.54B | Large cap | +6.36% |
| ARWR | Arrowhead Pharmaceuticals, Inc. | $12.52B | Large cap | +329.16% |
| CYTK | Cytokinetics, Incorporated | $10.77B | Large cap | +109.29% |
| IONS | Ionis Pharmaceuticals, Inc. | $10.56B | Large cap | +52.01% |
| KYMR | Kymera Therapeutics, Inc. | $10.53B | Large cap | +202.82% |
| ABVX.PA | ABIVAX Société Anonyme | $10.42B | Large cap | +42.59% |
| 0JDI.L | Ionis Pharmaceuticals, Inc. | $10.40B | Large cap | +49.07% |
| PTGX | Protagonist Therapeutics, Inc. | $10.01B | Large cap | +164.75% |
| 0HNC.L | BioMarin Pharmaceutical Inc. | $9.93B | Mid cap | +12.68% |
| CRNX | Crinetics Pharmaceuticals, Inc. | $9.00B | Mid cap | +182.02% |
| IMVT | Immunovant, Inc. | $8.95B | Mid cap | +192.14% |
| 4151.T | Kyowa Kirin Co., Ltd. | $8.51B | Mid cap | +3.09% |
| RYTM | Rhythm Pharmaceuticals, Inc. | $7.78B | Mid cap | +14.26% |
| SRRK | Scholar Rock Holding Corporation | $7.09B | Mid cap | +74.77% |
| 0HI3.L | Arrowhead Pharmaceuticals, Inc. | $6.98B | Mid cap | +317.86% |
| ORKA | Oruka Therapeutics, Inc. | $6.74B | Mid cap | +601.72% |
| LQDA | Liquidia Corporation | $6.31B | Mid cap | +168.53% |
| KNSA | Kiniksa Pharmaceuticals International, plc | $6.17B | Mid cap | +142.74% |
| PTCT | PTC Therapeutics, Inc. Common Stock | $6.13B | Mid cap | +49.03% |
| DNTH | Dianthus Therapeutics, Inc. | $6.13B | Mid cap | +378.90% |
| TVTX | Travere Therapeutics, Inc. | $6.06B | Mid cap | +288.91% |
| MIRM | Mirum Pharmaceuticals, Inc. | $5.98B | Mid cap | +36.58% |
| GRFS | Grifols, S.A. | $5.44B | Mid cap | −17.62% |
| DYN | Dyne Therapeutics, Inc. | $4.92B | Mid cap | +102.62% |
| EWTX | Edgewise Therapeutics, Inc. | $4.80B | Mid cap | +209.16% |
| AMLX | Amylyx Pharmaceuticals, Inc. | $4.17B | Mid cap | +340.71% |
| MANE | Veradermics, Incorporated | $4.11B | Mid cap | +171.13% |
| CAMX.ST | Camurus AB (publ) | $3.87B | Mid cap | −14.52% |
| CPRX | Catalyst Pharmaceuticals Inc | $3.85B | Mid cap | +52.72% |
| CLDX | Celldex Therapeutics, Inc. | $3.24B | Mid cap | +87.96% |
| IRON | Disc Medicine, Inc. | $3.16B | Mid cap | +38.71% |
| ARQT | Arcutis Biotherapeutics, Inc. | $3.12B | Mid cap | +58.44% |
| 0RD1.L | Camurus AB (publ) | $2.84B | Mid cap | −13.50% |
| VRDN | Viridian Therapeutics, Inc. | $2.74B | Mid cap | +30.21% |
| PHVS | Pharvaris N.V. | $2.62B | Mid cap | +57.76% |
| RARE | Ultragenyx Pharmaceutical Inc. | $2.61B | Mid cap | −9.60% |
| TRVI | Trevi Therapeutics, Inc. | $2.56B | Mid cap | +146.57% |
| VERA | Vera Therapeutics, Inc. | $2.54B | Mid cap | +65.32% |
| NKTR | Nektar Therapeutics | $2.52B | Mid cap | +176.20% |
| BCRX | BioCryst Pharmaceuticals, Inc. | $2.51B | Mid cap | +17.16% |
| MLYS | Mineralys Therapeutics, Inc. | $2.32B | Mid cap | +78.70% |
| 009420.KS | Hanall Biopharma Co., Ltd. | $2.32B | Mid cap | +108.57% |
| COAG | Hemab Therapeutics Holdings, Inc. | $2.32B | Mid cap | +46.03% |
| AUPH | Aurinia Pharmaceuticals Inc. | $2.23B | Mid cap | +38.03% |
| ZBIO | Zenas BioPharma, Inc. | $2.20B | Mid cap | +103.93% |
| STOK | Stoke Therapeutics, Inc. | $2.16B | Mid cap | +77.32% |
| ADMA | ADMA Biologics, Inc. | $2.14B | Mid cap | −42.86% |
| PVLA | Palvella Therapeutics, Inc. | $2.14B | Mid cap | +195.71% |
| NEU.AX | Neuren Pharmaceuticals Limited | $2.08B | Mid cap | +18.47% |
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.