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Rare disease stocks
We cover 136 companies grouped under the Rare disease theme. The median market value in the group is $1.46B. Across the group, the median move over the past trading day is −0.55%. 68% of the 130 with a 200-day average trade above it. Price change over the past year runs from −33.3% to +228.1% across the middle 80 percent of the group, with a median of +48.6%.
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Table
136 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 |
| SEPN | Septerna, Inc. | $40.25 | +18.49% | +43.85% | +218.81% | Nov 9, 2026 |
| ETON | Eton Pharmaceuticals, Inc. | $62.62 | +34.20% | +285.12% | +262.38% | Nov 12, 2026 |
| ANIP | ANI Pharmaceuticals, Inc. | $74.56 | −8.00% | −4.93% | −18.85% | Nov 6, 2026 |
| 0L35.L | Sarepta Therapeutics, Inc. | $18.51 | +1.60% | −13.25% | −0.06% | Nov 4, 2026 |
| MAZE | Maze Therapeutics, Inc. | $29.58 | +6.10% | −25.62% | +113.27% | Nov 5, 2026 |
| AVLN | Avalyn Pharma Inc. | $37.04 | +33.29% | +25.60% | +25.60% | Nov 16, 2026 |
| FBRX | Forte Biosciences, Inc. | $76.99 | +0.44% | +226.09% | +589.87% | Nov 9, 2026 |
| XERS | Xeris Biopharma Holdings, Inc. | $8.68 | +5.34% | +18.10% | +8.64% | Nov 5, 2026 |
| TRAX | First Tracks Biotherapeutics, Inc. | $44.19 | +5.59% | +132.58% | +132.58% | Nov 5, 2026 |
| TYRA | Tyra Biosciences, Inc. | $26.22 | −15.09% | −2.13% | +103.57% | Nov 4, 2026 |
| 4516.T | Nippon Shinyaku Co., Ltd. | 3,588.00 JPY | +1.07% | −34.41% | +19.48% | Nov 13, 2026 |
| 0LIF.L | Ultragenyx Pharmaceutical Inc. | $26.19 | +1.39% | +12.52% | −13.39% | Nov 3, 2026 |
| OMER | Omeros Corporation | $18.22 | +61.52% | +11.23% | +328.71% | Nov 12, 2026 |
| JBIO | Jade Biosciences, Inc. | $21.33 | +8.27% | +50.64% | +168.30% | Nov 12, 2026 |
| MLTX | MoonLake Immunotherapeutics | $15.62 | −16.52% | +30.71% | −71.82% | Nov 5, 2026 |
| 0HB0.L | Agios Pharmaceuticals, Inc. | $34.05 | +0.18% | +26.06% | −13.00% | Oct 29, 2026 |
| SVRA | Savara Inc. | $5.61 | +5.25% | −0.53% | +70.52% | Nov 11, 2026 |
| AVTX | Avalo Therapeutics, Inc. | $20.55 | +5.28% | +20.74% | +112.95% | Nov 12, 2026 |
| 006280.KS | GC Biopharma Corp. | 127,100.00 KRW | +12.28% | −19.46% | −2.60% | Nov 2, 2026 |
| WVE | Wave Life Sciences, Inc. | $5.23 | −11.21% | −67.21% | −48.12% | Nov 9, 2026 |
| DSGN | Design Therapeutics, Inc. | $15.59 | +31.34% | +71.32% | +194.71% | Nov 5, 2026 |
| ANNX | Annexon, Inc. | $4.99 | +0.40% | +6.40% | +136.49% | Nov 9, 2026 |
| DBVT | DBV Technologies S.A. | $14.52 | +7.56% | −23.78% | +53.00% | Oct 28, 2026 |
| DBV.PA | DBV Technologies S.A. | 2.53 EUR | +5.42% | −22.51% | +60.13% | Oct 28, 2026 |
| SLN | Silence Therapeutics plc | $13.53 | +17.14% | +120.00% | +160.19% | Nov 5, 2026 |
| GNFT.PA | Genfit S.A. | 14.30 EUR | +5.30% | +172.38% | +310.45% | Sep 29, 2026 |
| STTK | Shattuck Labs, Inc. | $7.90 | +15.84% | +112.94% | +690.00% | Nov 12, 2026 |
| IRWD | Ironwood Pharmaceuticals, Inc. | $4.29 | +12.89% | +0.47% | +240.48% | Nov 9, 2026 |
| GYRE | Gyre Therapeutics, Inc. | $6.65 | +11.02% | −2.35% | −18.00% | Nov 5, 2026 |
| TECX | Tectonic Therapeutic, Inc. | $35.97 | +20.70% | +72.02% | +35.43% | Nov 5, 2026 |
| ZVRA | Zevra Therapeutics, Inc. | $11.74 | +20.66% | +40.77% | +25.43% | Nov 4, 2026 |
| AQST | Aquestive Therapeutics, Inc. | $5.39 | +43.35% | −16.43% | +40.00% | Nov 4, 2026 |
| CRMD | CorMedix Inc. | $8.39 | +14.15% | −31.00% | −40.66% | Nov 16, 2026 |
| 0KBU.L | Omeros Corporation | $18.44 | −4.75% | +11.14% | +332.86% | Nov 12, 2026 |
| ZURA | Zura Bio Limited | $5.99 | +8.12% | +7.93% | +177.31% | Nov 12, 2026 |
| SPRY | ARS Pharmaceuticals, Inc. | $6.03 | +6.35% | −47.52% | −55.13% | Nov 10, 2026 |
| EPRX | Eupraxia Pharmaceuticals Inc. | $7.43 | +21.80% | −1.85% | +40.72% | Nov 3, 2026 |
| EPRX.TO | Eupraxia Pharmaceuticals Inc. | 10.28 CAD | +20.23% | −1.72% | +43.78% | Nov 3, 2026 |
| RZLT | Rezolute, Inc. | $5.04 | +2.44% | +132.79% | −30.00% | Sep 16, 2026 |
| KMDA | Kamada Ltd. Ordinary Shares | $8.38 | +18.70% | +22.82% | +21.09% | Nov 9, 2026 |
| AVIR | Atea Pharmaceuticals, Inc. | $5.51 | +22.17% | +58.33% | +53.06% | Nov 11, 2026 |
| LRMR | Larimar Therapeutics, Inc. | $4.17 | +8.88% | +20.17% | +6.92% | Nov 5, 2026 |
| XFOR | X4 Pharmaceuticals, Inc. | $4.17 | +6.11% | +7.20% | −1.42% | Nov 5, 2026 |
| ENTA | Enanta Pharmaceuticals, Inc. | $14.15 | +12.21% | −1.87% | +65.89% | Nov 16, 2026 |
| 4552.T | JCR Pharmaceuticals Co., Ltd. | 531.00 JPY | +2.71% | −21.85% | −9.63% | Oct 29, 2026 |
| UPB | Upstream Bio, Inc. | $7.13 | +11.41% | −73.66% | −57.31% | Nov 4, 2026 |
| IFRX | InflaRx N.V. | $2.42 | +43.20% | +122.02% | +142.00% | Nov 9, 2026 |
| IKT | Inhibikase Therapeutics, Inc. | $2.49 | +7.79% | +27.69% | +39.11% | Nov 13, 2026 |
| CUV.AX | Clinuvel Pharmaceuticals Limited | 9.36 AUD | −2.40% | −26.36% | −24.94% | Aug 27, 2026 |
| PRQR | ProQR Therapeutics N.V. | $2.35 | +33.52% | +18.09% | +6.33% | Nov 5, 2026 |
| TRDA | Entrada Therapeutics, Inc. | $7.00 | +20.07% | −30.35% | +23.24% | Nov 5, 2026 |
| FULC | Fulcrum Therapeutics, Inc. | $3.86 | +3.76% | −64.65% | −41.78% | Oct 29, 2026 |
| TTRX | Turn Therapeutics Inc. | $9.63 | +25.06% | +105.33% | +37.57% | Nov 12, 2026 |
| SION | Sionna Therapeutics, Inc. | $6.17 | −86.75% | −84.26% | −75.45% | Nov 5, 2026 |
| ELDN | Eledon Pharmaceuticals, Inc. | $3.35 | −1.47% | +106.79% | +29.84% | Nov 13, 2026 |
| IVVD | Invivyd, Inc. | $0.90 | +52.06% | −63.93% | +3.52% | Nov 5, 2026 |
| SLGL | Sol-Gel Technologies Ltd. | $79.43 | −4.38% | +88.45% | +261.87% | Nov 19, 2026 |
| XUP.DE | Genfit S.A. | 14.08 EUR | −1.81% | +164.41% | +166.82% | Sep 29, 2026 |
| AKBA | Akebia Therapeutics, Inc. | $0.91 | −28.62% | −41.52% | −69.88% | Nov 9, 2026 |
| CRDL | Cardiol Therapeutics Inc. | $2.14 | +78.33% | +115.29% | +91.07% | Nov 12, 2026 |
| CRDL.TO | Cardiol Therapeutics Inc. | 2.97 CAD | +74.71% | +118.38% | +90.38% | Nov 12, 2026 |
| PEPG | PepGen Inc. | $3.22 | +75.96% | −55.09% | +159.68% | Nov 11, 2026 |
| SIGA | SIGA Technologies, Inc. | $3.07 | −8.08% | −44.65% | −57.93% | Nov 5, 2026 |
| KROS | Keros Therapeutics, Inc. | $11.12 | +10.43% | −40.02% | −28.35% | Nov 4, 2026 |
| ARMP | Armata Pharmaceuticals, Inc. | $5.45 | +15.22% | −16.92% | +123.36% | Nov 11, 2026 |
| MSCL.TO | Satellos Bioscience Inc. | 13.08 CAD | −2.10% | +55.71% | +81.67% | Nov 12, 2026 |
| PLX | Protalix BioTherapeutics, Inc. | $2.48 | +6.44% | +42.53% | +55.00% | Nov 12, 2026 |
| MSLE | Satellos Bioscience Inc. | $9.36 | −1.06% | +50.87% | +77.27% | Nov 12, 2026 |
| 0AB6.L | Hyloris Pharmaceuticals SA | 4.38 EUR | −1.79% | −38.83% | −33.23% | Sep 24, 2026 |
| MDWD | MediWound Ltd. | $12.70 | −13.13% | −31.05% | −25.29% | Nov 19, 2026 |
| AARD | Aardvark Therapeutics, Inc. | $6.81 | +6.07% | −48.37% | −24.25% | Nov 12, 2026 |
| MIST | Milestone Pharmaceuticals Inc. | $1.18 | +10.28% | −42.44% | −33.33% | Nov 12, 2026 |
| INO | Inovio Pharmaceuticals, Inc. | $1.30 | +26.21% | −23.08% | −47.15% | Nov 10, 2026 |
| CPIX | Cumberland Pharmaceuticals Inc. | $8.97 | +69.86% | +125.38% | +163.82% | Nov 3, 2026 |
| 0H8T.L | Aclaris Therapeutics, Inc. | $6.14 | −6.69% | +105.92% | +237.38% | Nov 5, 2026 |
| RFL | Rafael Holdings, Inc. | $2.29 | +9.05% | +81.75% | +52.67% | Oct 28, 2026 |
| MDP.TO | Medexus Pharmaceuticals Inc. | 5.18 CAD | +7.02% | +67.64% | +66.56% | Nov 11, 2026 |
| PYPD | PolyPid Ltd. | $5.11 | +36.27% | +17.47% | +49.42% | Nov 11, 2026 |
| PTHS | Pelthos Therapeutics Inc. | $26.78 | −3.56% | −9.07% | +12.90% | 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.