Free tool
Rare disease stocks
We cover 92 companies grouped under the Rare disease theme. The median market value in the group is $765.04M. Across the group, the median move over the past trading day is +2.01%. 69% of the 87 with a 200-day average trade above it. Price change over the past year runs from −31.9% to +268.6% across the middle 80 percent of the group, with a median of +49.5%.
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
92 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return | Next report |
|---|---|---|---|---|---|
| 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 |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $1.95B | Small cap | −9.02% | Oct 29, 2026 |
| SEPN | Septerna, Inc. | $1.93B | Small cap | +245.32% | Nov 9, 2026 |
| ETON | Eton Pharmaceuticals, Inc. | $1.80B | Small cap | +268.93% | Nov 12, 2026 |
| ANIP | ANI Pharmaceuticals, Inc. | $1.74B | Small cap | −16.24% | Nov 6, 2026 |
| AVLN | Avalyn Pharma Inc. | $1.69B | Small cap | +29.54% | Nov 16, 2026 |
| 0L35.L | Sarepta Therapeutics, Inc. | $1.69B | Small cap | +4.63% | Nov 4, 2026 |
| MAZE | Maze Therapeutics, Inc. | $1.69B | Small cap | +128.55% | Nov 5, 2026 |
| FBRX | Forte Biosciences, Inc. | $1.63B | Small cap | +580.25% | Nov 9, 2026 |
| TRAX | First Tracks Biotherapeutics, Inc. | $1.61B | Small cap | +137.42% | Nov 5, 2026 |
| XERS | Xeris Biopharma Holdings, Inc. | $1.59B | Small cap | +12.48% | Nov 5, 2026 |
| TYRA | Tyra Biosciences, Inc. | $1.59B | Small cap | +122.42% | Nov 4, 2026 |
| 0LIF.L | Ultragenyx Pharmaceutical Inc. | $1.40B | Small cap | −11.63% | Nov 3, 2026 |
| OMER | Omeros Corporation | $1.40B | Small cap | +343.22% | Nov 12, 2026 |
| JBIO | Jade Biosciences, Inc. | $1.39B | Small cap | +176.46% | Nov 12, 2026 |
| MLTX | MoonLake Immunotherapeutics | $1.32B | Small cap | −71.52% | Nov 5, 2026 |
| 0HB0.L | Agios Pharmaceuticals, Inc. | $1.19B | Small cap | −14.25% | Oct 29, 2026 |
| SVRA | Savara Inc. | $1.15B | Small cap | +78.34% | Nov 11, 2026 |
| ANNX | Annexon, Inc. | $976.33M | Small cap | +139.53% | Nov 9, 2026 |
| DBVT | DBV Technologies S.A. | $940.80M | Small cap | +52.91% | Oct 28, 2026 |
| DBV.PA | DBV Technologies S.A. | $928.30M | Small cap | +54.11% | Oct 28, 2026 |
| SLN | Silence Therapeutics plc | $849.65M | Small cap | +170.16% | Nov 5, 2026 |
| GNFT.PA | Genfit S.A. | $836.50M | Small cap | +293.99% | Sep 29, 2026 |
| STTK | Shattuck Labs, Inc. | $792.87M | Small cap | +710.87% | Nov 12, 2026 |
| TECX | Tectonic Therapeutic, Inc. | $737.21M | Small cap | +53.48% | Nov 5, 2026 |
| ZVRA | Zevra Therapeutics, Inc. | $714.98M | Small cap | +28.63% | Nov 4, 2026 |
| GYRE | Gyre Therapeutics, Inc. | $682.86M | Small cap | −18.54% | Nov 5, 2026 |
| CRMD | CorMedix Inc. | $667.20M | Small cap | −37.65% | Nov 16, 2026 |
| ZURA | Zura Bio Limited | $605.64M | Small cap | +208.29% | Nov 12, 2026 |
| AQST | Aquestive Therapeutics, Inc. | $602.46M | Small cap | +31.61% | Nov 4, 2026 |
| 0KBU.L | Omeros Corporation | $575.28M | Small cap | +341.05% | Nov 12, 2026 |
| SPRY | ARS Pharmaceuticals, Inc. | $568.84M | Small cap | −57.66% | Nov 10, 2026 |
| KMDA | Kamada Ltd. Ordinary Shares | $497.90M | Small cap | +26.12% | Nov 9, 2026 |
| EPRX | Eupraxia Pharmaceuticals Inc. | $490.26M | Small cap | +41.87% | Nov 3, 2026 |
| EPRX.TO | Eupraxia Pharmaceuticals Inc. | $487.17M | Small cap | +41.67% | Nov 3, 2026 |
| RZLT | Rezolute, Inc. | $482.42M | Small cap | −28.43% | Sep 16, 2026 |
| AVIR | Atea Pharmaceuticals, Inc. | $441.93M | Small cap | +48.36% | Nov 11, 2026 |
| LRMR | Larimar Therapeutics, Inc. | $432.15M | Small cap | +11.83% | Nov 5, 2026 |
| XFOR | X4 Pharmaceuticals, Inc. | $412.45M | Small cap | +4.79% | Nov 5, 2026 |
| ENTA | Enanta Pharmaceuticals, Inc. | $410.67M | Small cap | +62.59% | Nov 16, 2026 |
| 4552.T | JCR Pharmaceuticals Co., Ltd. | $397.97M | Small cap | −7.40% | Oct 29, 2026 |
| UPB | Upstream Bio, Inc. | $393.26M | Small cap | −57.08% | Nov 4, 2026 |
| CUV.AX | Clinuvel Pharmaceuticals Limited | $381.21M | Small cap | −21.84% | Aug 27, 2026 |
| IKT | Inhibikase Therapeutics, Inc. | $348.84M | Small cap | +42.05% | Nov 13, 2026 |
| PRQR | ProQR Therapeutics N.V. | $335.86M | Small cap | +5.78% | Nov 5, 2026 |
| TRDA | Entrada Therapeutics, Inc. | $296.70M | Small cap | +35.35% | Nov 5, 2026 |
| TTRX | Turn Therapeutics Inc. | $295.48M | Small cap | +39.00% | Nov 12, 2026 |
| FULC | Fulcrum Therapeutics, Inc. | $294.51M | Small cap | −39.78% | Oct 29, 2026 |
| SION | Sionna Therapeutics, Inc. | $282.58M | Small cap | −74.02% | Nov 5, 2026 |
| ELDN | Eledon Pharmaceuticals, Inc. | $281.98M | Small cap | +35.02% | Nov 13, 2026 |
| IVVD | Invivyd, Inc. | $277.81M | Small cap | +67.62% | Nov 5, 2026 |
| SLGL | Sol-Gel Technologies Ltd. | $265.20M | Small cap | +289.14% | Nov 19, 2026 |
| XUP.DE | Genfit S.A. | $258.22M | Small cap | +166.82% | Sep 29, 2026 |
| CRDL | Cardiol Therapeutics Inc. | $233.65M | Small cap | +75.44% | Nov 12, 2026 |
| CRDL.TO | Cardiol Therapeutics Inc. | $233.07M | Small cap | +72.50% | Nov 12, 2026 |
| SIGA | SIGA Technologies, Inc. | $211.24M | Small cap | −60.50% | Nov 5, 2026 |
| ARMP | Armata Pharmaceuticals, Inc. | $206.73M | Small cap | +113.85% | Nov 11, 2026 |
| MSLE | Satellos Bioscience Inc. | $200.92M | Small cap | +82.17% | Nov 12, 2026 |
| MSCL.TO | Satellos Bioscience Inc. | $199.37M | Small cap | +80.56% | Nov 12, 2026 |
| PLX | Protalix BioTherapeutics, Inc. | $193.37M | Small cap | +50.62% | Nov 12, 2026 |
| 0AB6.L | Hyloris Pharmaceuticals SA | $185.32M | Small cap | −32.01% | Sep 24, 2026 |
| AARD | Aardvark Therapeutics, Inc. | $158.67M | Small cap | −19.80% | Nov 12, 2026 |
| MIST | Milestone Pharmaceuticals Inc. | $144.42M | Small cap | −31.36% | Nov 12, 2026 |
| CPIX | Cumberland Pharmaceuticals Inc. | $130.35M | Small cap | +166.87% | Nov 3, 2026 |
| INO | Inovio Pharmaceuticals, Inc. | $129.25M | Small cap | −33.85% | Nov 10, 2026 |
| 0H8T.L | Aclaris Therapeutics, Inc. | $122.82M | Small cap | +265.21% | Nov 5, 2026 |
| MDP.TO | Medexus Pharmaceuticals Inc. | $116.99M | Small cap | +80.70% | Nov 11, 2026 |
| RFL | Rafael Holdings, Inc. | $113.88M | Small cap | +52.38% | Oct 28, 2026 |
| PTHS | Pelthos Therapeutics Inc. | $104.13M | Small cap | +20.41% | Nov 12, 2026 |
| PYPD | PolyPid Ltd. | $101.25M | Small cap | +47.83% | Nov 11, 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.