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Rare disease stocks
We cover 52 companies grouped under the Rare disease theme. The median market value in the group is $3.62B. Across the group, the median move over the past trading day is +1.20%. 59% of the 49 with a 200-day average trade above it. Price change over the past year runs from −75.5% to +200.2% across the middle 80 percent of the group, with a median of +38.4%.
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SaaSAI applicationsCybersecuritySemiconductor designSemiconductor equipmentFintech softwarePayment processingEcommerce marketplacesStreaming mediaGaming softwareOncologyRare diseaseMedical devicesCell therapyDiagnosticsRegional banksAsset managementGold miningCryptocurrency and digital assetsSpecialty chemicalsAll stocks
Table
52 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return | Next report |
|---|---|---|---|---|---|
| VX1.DE | Vertex Pharmaceuticals Incorporated | $140.77B | Large cap | +40.14% | Nov 2, 2026 |
| VRTX | Vertex Pharmaceuticals Incorporated | $140.04B | Large cap | +42.14% | Nov 2, 2026 |
| 0QZU.L | Vertex Pharmaceuticals Incorporated | $112.52B | Large cap | +42.39% | Nov 2, 2026 |
| ARGX | argenx SE | $63.78B | Large cap | +50.52% | Oct 22, 2026 |
| 0HD2.L | Alnylam Pharmaceuticals, Inc. | $35.05B | Large cap | −46.85% | Oct 29, 2026 |
| ALNY | Alnylam Pharmaceuticals, Inc. | $32.12B | Large cap | −45.88% | Oct 29, 2026 |
| DUL.DE | Alnylam Pharmaceuticals, Inc. | $31.87B | Large cap | −11.75% | Oct 29, 2026 |
| INSM | Insmed Incorporated | $27.07B | Large cap | −3.45% | Oct 29, 2026 |
| BBIO | BridgeBio Pharma, Inc. | $15.85B | Large cap | +70.01% | Oct 28, 2026 |
| 0JAV.L | Insmed Incorporated | $12.89B | Large cap | −6.25% | Oct 29, 2026 |
| BMRN | BioMarin Pharmaceutical Inc. | $12.80B | Large cap | +13.03% | Oct 26, 2026 |
| CYTK | Cytokinetics, Incorporated | $10.77B | Large cap | +109.29% | Nov 5, 2026 |
| KYMR | Kymera Therapeutics, Inc. | $10.53B | Large cap | +202.82% | Nov 3, 2026 |
| ABVX.PA | ABIVAX Société Anonyme | $10.42B | Large cap | +42.59% | Sep 21, 2026 |
| PTGX | Protagonist Therapeutics, Inc. | $10.01B | Large cap | +164.75% | Nov 5, 2026 |
| 0HNC.L | BioMarin Pharmaceutical Inc. | $9.93B | Mid cap | +12.68% | Oct 26, 2026 |
| 4151.T | Kyowa Kirin Co., Ltd. | $8.51B | Mid cap | +3.09% | Nov 2, 2026 |
| RYTM | Rhythm Pharmaceuticals, Inc. | $7.78B | Mid cap | +14.26% | Nov 3, 2026 |
| ORKA | Oruka Therapeutics, Inc. | $6.74B | Mid cap | +601.72% | Nov 11, 2026 |
| LQDA | Liquidia Corporation | $6.31B | Mid cap | +168.53% | Nov 3, 2026 |
| KNSA | Kiniksa Pharmaceuticals International, plc | $6.17B | Mid cap | +142.74% | Oct 27, 2026 |
| TVTX | Travere Therapeutics, Inc. | $6.06B | Mid cap | +288.91% | Oct 29, 2026 |
| MIRM | Mirum Pharmaceuticals, Inc. | $5.98B | Mid cap | +36.58% | Nov 4, 2026 |
| EWTX | Edgewise Therapeutics, Inc. | $4.80B | Mid cap | +209.16% | Nov 5, 2026 |
| AMLX | Amylyx Pharmaceuticals, Inc. | $4.17B | Mid cap | +340.71% | Nov 5, 2026 |
| MANE | Veradermics, Incorporated | $4.11B | Mid cap | +171.13% | Nov 12, 2026 |
| ARQT | Arcutis Biotherapeutics, Inc. | $3.12B | Mid cap | +58.44% | Oct 27, 2026 |
| VRDN | Viridian Therapeutics, Inc. | $2.74B | Mid cap | +30.21% | Nov 5, 2026 |
| PHVS | Pharvaris N.V. | $2.62B | Mid cap | +57.76% | Nov 12, 2026 |
| RARE | Ultragenyx Pharmaceutical Inc. | $2.61B | Mid cap | −9.60% | Nov 3, 2026 |
| VERA | Vera Therapeutics, Inc. | $2.54B | Mid cap | +65.32% | Nov 5, 2026 |
| NKTR | Nektar Therapeutics | $2.52B | Mid cap | +176.20% | Nov 12, 2026 |
| BCRX | BioCryst Pharmaceuticals, Inc. | $2.51B | Mid cap | +17.16% | Nov 2, 2026 |
| MLYS | Mineralys Therapeutics, Inc. | $2.32B | Mid cap | +78.70% | Nov 10, 2026 |
| COAG | Hemab Therapeutics Holdings, Inc. | $2.32B | Mid cap | +46.03% | Nov 16, 2026 |
| ZBIO | Zenas BioPharma, Inc. | $2.20B | Mid cap | +103.93% | Nov 12, 2026 |
| STOK | Stoke Therapeutics, Inc. | $2.16B | Mid cap | +77.32% | Nov 3, 2026 |
| ADMA | ADMA Biologics, Inc. | $2.14B | Mid cap | −42.86% | Nov 4, 2026 |
| GOSS | Gossamer Bio, Inc. | $99.97M | Micro cap | −91.33% | Nov 4, 2026 |
| SPRO | Spero Therapeutics, Inc. | $70.76M | Micro cap | −33.51% | Nov 12, 2026 |
| ATYR | aTyr Pharma, Inc. | $55.91M | Micro cap | −88.95% | Nov 5, 2026 |
| SCYX | SCYNEXIS, Inc. | $51.64M | Micro cap | +514.20% | Nov 4, 2026 |
| MCRB | Seres Therapeutics, Inc. | $46.81M | Micro cap | −74.00% | Nov 4, 2026 |
| MREO | Mereo BioPharma Group plc | $44.97M | Micro cap | −83.82% | Nov 9, 2026 |
| QNCX | Quince Therapeutics, Inc. | $34.05M | Micro cap | +95.76% | Nov 12, 2026 |
| JSPR | Jasper Therapeutics, Inc. | $23.96M | Micro cap | −75.67% | Nov 10, 2026 |
| TVRD | Tvardi Therapeutics, Inc. | $23.74M | Micro cap | −93.77% | Nov 12, 2026 |
| RVX.TO | Resverlogix Corp. | $23.62M | Micro cap | +5.00% | Nov 12, 2026 |
| INM | InMed Pharmaceuticals Inc. | $7.69M | Micro cap | −35.43% | Sep 23, 2026 |
| JAGX | Jaguar Health, Inc. | $4.78M | Micro cap | −98.52% | Nov 12, 2026 |
| XRTX | XORTX Therapeutics Inc. | $4.11M | Micro cap | −44.74% | Nov 12, 2026 |
| APLI.TO | Appili Therapeutics Inc. | $1.44M | Micro cap | −25.00% | Nov 12, 2026 |
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.