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Rare disease stocks
We cover 71 companies grouped under the Rare disease theme. The median market value in the group is $4.80B. Across the group, the median move over the past trading day is −0.26%. 68% of the 68 with a 200-day average trade above it. Price change over the past year runs from −47.4% to +206.5% across the middle 80 percent of the group, with a median of +46.0%.
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Table
71 of 268 companies match these filters.Clear filters
| Ticker | Company | Price | 1M return | YTD return | 1Y return | Next report |
|---|---|---|---|---|---|---|
| VX1.DE | Vertex Pharmaceuticals Incorporated | 475.00 EUR | +10.21% | +23.55% | +41.64% | Nov 2, 2026 |
| VRTX | Vertex Pharmaceuticals Incorporated | $547.29 | +11.60% | +21.05% | +40.66% | Nov 2, 2026 |
| 0QZU.L | Vertex Pharmaceuticals Incorporated | $549.02 | +0.13% | +22.11% | +40.77% | Nov 2, 2026 |
| ARGX | argenx SE | $1,047.10 | +20.05% | +24.66% | +47.90% | Oct 22, 2026 |
| 0HD2.L | Alnylam Pharmaceuticals, Inc. | $241.82 | +0.67% | −39.23% | −46.60% | Oct 29, 2026 |
| DUL.DE | Alnylam Pharmaceuticals, Inc. | 204.00 EUR | −19.59% | −40.19% | −10.21% | Oct 29, 2026 |
| ALNY | Alnylam Pharmaceuticals, Inc. | $239.00 | −17.13% | −40.28% | −47.38% | Oct 29, 2026 |
| INSM | Insmed Incorporated | $124.39 | +19.46% | −29.77% | −7.54% | Oct 29, 2026 |
| ROIV | Roivant Sciences Ltd. | $36.75 | +8.60% | +67.58% | +206.51% | Nov 9, 2026 |
| JAZZ | Jazz Pharmaceuticals plc | $249.29 | −2.97% | +43.97% | +100.35% | Nov 4, 2026 |
| BBIO | BridgeBio Pharma, Inc. | $79.37 | −7.22% | +1.45% | +60.05% | Oct 28, 2026 |
| CORT | Corcept Therapeutics Incorporated | $119.80 | +27.19% | +213.61% | +71.81% | Nov 4, 2026 |
| 0JAV.L | Insmed Incorporated | $125.00 | +0.43% | −28.28% | −7.52% | Oct 29, 2026 |
| BMRN | BioMarin Pharmaceutical Inc. | $65.61 | +5.70% | +10.36% | +11.85% | Oct 26, 2026 |
| REC.MI | Recordati Industria Chimica e Farmaceutica S.p.A. | 52.70 EUR | +2.53% | +10.68% | +1.80% | Nov 10, 2026 |
| ARWR | Arrowhead Pharmaceuticals, Inc. | $89.12 | +3.89% | +31.46% | +308.99% | Nov 24, 2026 |
| CYTK | Cytokinetics, Incorporated | $79.20 | −0.31% | +28.30% | +109.36% | Nov 5, 2026 |
| IONS | Ionis Pharmaceuticals, Inc. | $63.36 | +14.58% | −20.47% | +48.21% | Nov 4, 2026 |
| KYMR | Kymera Therapeutics, Inc. | $125.55 | +18.26% | +72.55% | +197.09% | Nov 3, 2026 |
| ABVX.PA | ABIVAX Société Anonyme | 102.30 EUR | −5.63% | −11.04% | +48.91% | Sep 21, 2026 |
| 0HNC.L | BioMarin Pharmaceutical Inc. | $66.19 | +0.05% | +11.54% | +15.03% | Oct 26, 2026 |
| PTGX | Protagonist Therapeutics, Inc. | $151.50 | +10.71% | +73.78% | +146.62% | Nov 5, 2026 |
| IMVT | Immunovant, Inc. | $43.89 | +12.83% | +69.39% | +189.13% | Nov 9, 2026 |
| CRNX | Crinetics Pharmaceuticals, Inc. | $84.84 | +1.17% | +81.55% | +179.08% | Nov 5, 2026 |
| 4151.T | Kyowa Kirin Co., Ltd. | 2,613.00 JPY | −3.33% | +3.94% | +4.01% | Nov 2, 2026 |
| RYTM | Rhythm Pharmaceuticals, Inc. | $111.87 | +3.54% | +6.53% | +9.35% | Nov 3, 2026 |
| SRRK | Scholar Rock Holding Corporation | $60.04 | +21.61% | +40.28% | +74.18% | Nov 12, 2026 |
| ORKA | Oruka Therapeutics, Inc. | $99.44 | +9.94% | +251.75% | +555.07% | Nov 11, 2026 |
| LQDA | Liquidia Corporation | $71.36 | −17.81% | +127.26% | +154.86% | Nov 3, 2026 |
| TVTX | Travere Therapeutics, Inc. | $67.12 | +20.05% | +69.84% | +284.86% | Oct 29, 2026 |
| DNTH | Dianthus Therapeutics, Inc. | $110.76 | +9.01% | +179.13% | +356.93% | Nov 5, 2026 |
| PTCT | PTC Therapeutics, Inc. Common Stock | $72.69 | −3.58% | −5.28% | +46.02% | Nov 5, 2026 |
| KNSA | Kiniksa Pharmaceuticals International, plc | $78.68 | −0.96% | +87.31% | +133.47% | Oct 27, 2026 |
| MIRM | Mirum Pharmaceuticals, Inc. | $98.51 | −12.22% | +26.18% | +31.96% | Nov 4, 2026 |
| DYN | Dyne Therapeutics, Inc. | $26.36 | +1.78% | +42.49% | +93.26% | Nov 5, 2026 |
| EWTX | Edgewise Therapeutics, Inc. | $44.38 | +9.99% | +86.86% | +207.13% | Nov 5, 2026 |
| MANE | Veradermics, Incorporated | $102.80 | −7.84% | +172.32% | +172.32% | Nov 12, 2026 |
| AMLX | Amylyx Pharmaceuticals, Inc. | $37.08 | +89.67% | +224.69% | +292.38% | Nov 5, 2026 |
| CAMX.ST | Camurus AB (publ) | 615.00 SEK | −1.68% | +0.49% | −15.35% | Nov 5, 2026 |
| CLDX | Celldex Therapeutics, Inc. | $40.57 | +14.02% | +50.20% | +79.51% | Nov 9, 2026 |
| IRON | Disc Medicine, Inc. | $82.67 | +10.74% | +4.53% | +37.90% | Nov 5, 2026 |
| ARQT | Arcutis Biotherapeutics, Inc. | $25.14 | −11.54% | −13.31% | +60.64% | Oct 27, 2026 |
| VRDN | Viridian Therapeutics, Inc. | $24.26 | +24.35% | −20.93% | +30.71% | Nov 5, 2026 |
| VERA | Vera Therapeutics, Inc. | $36.82 | +10.50% | −24.27% | +67.21% | Nov 5, 2026 |
| RARE | Ultragenyx Pharmaceutical Inc. | $26.44 | +1.77% | +12.03% | −12.10% | Nov 3, 2026 |
| PHVS | Pharvaris N.V. | $37.00 | +9.97% | +39.73% | +55.59% | Nov 12, 2026 |
| TRVI | Trevi Therapeutics, Inc. | $17.92 | −2.77% | +50.71% | +124.00% | Nov 12, 2026 |
| NKTR | Nektar Therapeutics | $73.77 | +4.24% | +69.90% | +151.95% | Nov 12, 2026 |
| BCRX | BioCryst Pharmaceuticals, Inc. | $9.83 | +0.92% | +31.07% | +15.38% | Nov 2, 2026 |
| COAG | Hemab Therapeutics Holdings, Inc. | $49.19 | +4.08% | +44.68% | +44.68% | Nov 16, 2026 |
| MLYS | Mineralys Therapeutics, Inc. | $25.89 | −3.93% | −27.50% | +70.22% | Nov 10, 2026 |
| PVLA | Palvella Therapeutics, Inc. | $154.33 | +8.04% | +53.90% | +173.39% | Nov 11, 2026 |
| AUPH | Aurinia Pharmaceuticals Inc. | $16.54 | +10.56% | +7.75% | +36.58% | Nov 9, 2026 |
| ADMA | ADMA Biologics, Inc. | $9.63 | +9.56% | −46.14% | −44.17% | Nov 4, 2026 |
| ZBIO | Zenas BioPharma, Inc. | $33.04 | +2.32% | −4.23% | +97.73% | Nov 12, 2026 |
| STOK | Stoke Therapeutics, Inc. | $33.09 | +12.59% | +7.09% | +71.45% | Nov 3, 2026 |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $33.99 | −5.56% | +25.06% | −13.45% | Oct 29, 2026 |
| GOSS | Gossamer Bio, Inc. | $0.17 | −15.17% | −93.96% | −92.93% | Nov 4, 2026 |
| SPRO | Spero Therapeutics, Inc. | $1.22 | +2.09% | −48.09% | −38.38% | Nov 12, 2026 |
| SCYX | SCYNEXIS, Inc. | $5.67 | +56.20% | +787.32% | +579.04% | Nov 4, 2026 |
| ATYR | aTyr Pharma, Inc. | $0.55 | +11.54% | −27.40% | −89.34% | Nov 5, 2026 |
| MCRB | Seres Therapeutics, Inc. | $4.76 | −0.83% | −68.58% | −74.79% | Nov 4, 2026 |
| MREO | Mereo BioPharma Group plc | $0.28 | −10.03% | −45.49% | −84.20% | Nov 9, 2026 |
| QNCX | Quince Therapeutics, Inc. | $31.31 | +61.64% | +4.37% | +100.71% | Nov 12, 2026 |
| RVX.TO | Resverlogix Corp. | 0.10 CAD | 0.00% | 0.00% | 0.00% | Nov 12, 2026 |
| JSPR | Jasper Therapeutics, Inc. | $0.72 | +13.43% | −60.98% | −75.24% | Nov 10, 2026 |
| TVRD | Tvardi Therapeutics, Inc. | $1.78 | −27.05% | −56.37% | −94.18% | Nov 12, 2026 |
| INM | InMed Pharmaceuticals Inc. | $1.43 | −14.37% | +27.68% | −36.44% | Sep 23, 2026 |
| JAGX | Jaguar Health, Inc. | $0.92 | −4.63% | −97.37% | −98.52% | Nov 12, 2026 |
| XRTX | XORTX Therapeutics Inc. | $2.22 | +8.20% | −22.18% | −43.99% | Nov 12, 2026 |
| APLI.TO | Appili Therapeutics Inc. | 0.02 CAD | +50.00% | −40.00% | −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.