Free tool
Rare disease stocks
We cover 57 companies grouped under the Rare disease theme. The median market value in the group is $6.31B. Across the group, the median move over the past trading day is 0.00%. 80% of the 54 with a 200-day average trade above it. Price change over the past year runs from −11.0% to +206.8% across the middle 80 percent of the group, with a median of +60.0%.
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
57 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return | Next report |
|---|---|---|---|---|---|
| VX1.DE | Vertex Pharmaceuticals Incorporated | $139.82B | Large cap | +41.64% | Nov 2, 2026 |
| VRTX | Vertex Pharmaceuticals Incorporated | $138.63B | Large cap | +40.66% | Nov 2, 2026 |
| 0QZU.L | Vertex Pharmaceuticals Incorporated | $112.52B | Large cap | +40.77% | Nov 2, 2026 |
| ARGX | argenx SE | $65.51B | Large cap | +47.90% | Oct 22, 2026 |
| 0HD2.L | Alnylam Pharmaceuticals, Inc. | $35.05B | Large cap | −46.60% | Oct 29, 2026 |
| DUL.DE | Alnylam Pharmaceuticals, Inc. | $32.28B | Large cap | −10.21% | Oct 29, 2026 |
| ALNY | Alnylam Pharmaceuticals, Inc. | $32.12B | Large cap | −47.38% | Oct 29, 2026 |
| INSM | Insmed Incorporated | $27.16B | Large cap | −7.54% | Oct 29, 2026 |
| ROIV | Roivant Sciences Ltd. | $26.55B | Large cap | +206.51% | Nov 9, 2026 |
| JAZZ | Jazz Pharmaceuticals plc | $16.89B | Large cap | +100.35% | Nov 4, 2026 |
| BBIO | BridgeBio Pharma, Inc. | $15.52B | Large cap | +60.05% | Oct 28, 2026 |
| CORT | Corcept Therapeutics Incorporated | $12.95B | Large cap | +71.81% | Nov 4, 2026 |
| 0JAV.L | Insmed Incorporated | $12.89B | Large cap | −7.52% | Oct 29, 2026 |
| BMRN | BioMarin Pharmaceutical Inc. | $12.70B | Large cap | +11.85% | Oct 26, 2026 |
| REC.MI | Recordati Industria Chimica e Farmaceutica S.p.A. | $12.57B | Large cap | +1.80% | Nov 10, 2026 |
| ARWR | Arrowhead Pharmaceuticals, Inc. | $12.52B | Large cap | +308.99% | Nov 24, 2026 |
| CYTK | Cytokinetics, Incorporated | $11.01B | Large cap | +109.36% | Nov 5, 2026 |
| IONS | Ionis Pharmaceuticals, Inc. | $10.56B | Large cap | +48.21% | Nov 4, 2026 |
| KYMR | Kymera Therapeutics, Inc. | $10.53B | Large cap | +197.09% | Nov 3, 2026 |
| ABVX.PA | ABIVAX Société Anonyme | $10.21B | Large cap | +48.91% | Sep 21, 2026 |
| 0HNC.L | BioMarin Pharmaceutical Inc. | $9.93B | Mid cap | +15.03% | Oct 26, 2026 |
| PTGX | Protagonist Therapeutics, Inc. | $9.80B | Mid cap | +146.62% | Nov 5, 2026 |
| IMVT | Immunovant, Inc. | $9.07B | Mid cap | +189.13% | Nov 9, 2026 |
| CRNX | Crinetics Pharmaceuticals, Inc. | $9.00B | Mid cap | +179.08% | Nov 5, 2026 |
| 4151.T | Kyowa Kirin Co., Ltd. | $8.51B | Mid cap | +4.01% | Nov 2, 2026 |
| RYTM | Rhythm Pharmaceuticals, Inc. | $7.78B | Mid cap | +9.35% | Nov 3, 2026 |
| SRRK | Scholar Rock Holding Corporation | $7.31B | Mid cap | +74.18% | Nov 12, 2026 |
| ORKA | Oruka Therapeutics, Inc. | $6.58B | Mid cap | +555.07% | Nov 11, 2026 |
| LQDA | Liquidia Corporation | $6.31B | Mid cap | +154.86% | Nov 3, 2026 |
| TVTX | Travere Therapeutics, Inc. | $6.24B | Mid cap | +284.86% | Oct 29, 2026 |
| DNTH | Dianthus Therapeutics, Inc. | $6.13B | Mid cap | +356.93% | Nov 5, 2026 |
| PTCT | PTC Therapeutics, Inc. Common Stock | $6.07B | Mid cap | +46.02% | Nov 5, 2026 |
| KNSA | Kiniksa Pharmaceuticals International, plc | $6.05B | Mid cap | +133.47% | Oct 27, 2026 |
| MIRM | Mirum Pharmaceuticals, Inc. | $5.98B | Mid cap | +31.96% | Nov 4, 2026 |
| DYN | Dyne Therapeutics, Inc. | $4.92B | Mid cap | +93.26% | Nov 5, 2026 |
| EWTX | Edgewise Therapeutics, Inc. | $4.80B | Mid cap | +207.13% | Nov 5, 2026 |
| MANE | Veradermics, Incorporated | $4.29B | Mid cap | +172.32% | Nov 12, 2026 |
| AMLX | Amylyx Pharmaceuticals, Inc. | $4.13B | Mid cap | +292.38% | Nov 5, 2026 |
| CAMX.ST | Camurus AB (publ) | $3.79B | Mid cap | −15.35% | Nov 5, 2026 |
| CLDX | Celldex Therapeutics, Inc. | $3.19B | Mid cap | +79.51% | Nov 9, 2026 |
| IRON | Disc Medicine, Inc. | $3.17B | Mid cap | +37.90% | Nov 5, 2026 |
| ARQT | Arcutis Biotherapeutics, Inc. | $3.12B | Mid cap | +60.64% | Oct 27, 2026 |
| VRDN | Viridian Therapeutics, Inc. | $2.75B | Mid cap | +30.71% | Nov 5, 2026 |
| VERA | Vera Therapeutics, Inc. | $2.65B | Mid cap | +67.21% | Nov 5, 2026 |
| RARE | Ultragenyx Pharmaceutical Inc. | $2.61B | Mid cap | −12.10% | Nov 3, 2026 |
| PHVS | Pharvaris N.V. | $2.60B | Mid cap | +55.59% | Nov 12, 2026 |
| TRVI | Trevi Therapeutics, Inc. | $2.55B | Mid cap | +124.00% | Nov 12, 2026 |
| NKTR | Nektar Therapeutics | $2.52B | Mid cap | +151.95% | Nov 12, 2026 |
| BCRX | BioCryst Pharmaceuticals, Inc. | $2.51B | Mid cap | +15.38% | Nov 2, 2026 |
| COAG | Hemab Therapeutics Holdings, Inc. | $2.30B | Mid cap | +44.68% | Nov 16, 2026 |
| MLYS | Mineralys Therapeutics, Inc. | $2.28B | Mid cap | +70.22% | Nov 10, 2026 |
| PVLA | Palvella Therapeutics, Inc. | $2.22B | Mid cap | +173.39% | Nov 11, 2026 |
| AUPH | Aurinia Pharmaceuticals Inc. | $2.20B | Mid cap | +36.58% | Nov 9, 2026 |
| ADMA | ADMA Biologics, Inc. | $2.16B | Mid cap | −44.17% | Nov 4, 2026 |
| ZBIO | Zenas BioPharma, Inc. | $2.15B | Mid cap | +97.73% | Nov 12, 2026 |
| STOK | Stoke Therapeutics, Inc. | $2.14B | Mid cap | +71.45% | Nov 3, 2026 |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $2.03B | Mid cap | −13.45% | Oct 29, 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.