Free tool
Rare disease stocks
We cover 38 companies grouped under the Rare disease theme. The median market value in the group is $6.53B. Across the group, the median move over the past trading day is +1.15%. 74% of the 35 with a 200-day average trade above it. Price change over the past year runs from −10.2% to +204.7% across the middle 80 percent of the group, with a median of +48.3%.
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
38 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 |
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.