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Rare disease stocks
We cover 146 companies grouped under the Rare disease theme. The median market value in the group is $834.78M. Across the group, the median move over the past trading day is +1.78%. 68% of the 139 with a 200-day average trade above it. Price change over the past year runs from −31.8% to +292.0% across the middle 80 percent of the group, with a median of +48.4%.
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Table
146 of 268 companies match these filters.Clear filters
| Ticker | Company | Price | 1M return | YTD return | 1Y return |
|---|---|---|---|---|---|
| 0HNC.L | BioMarin Pharmaceutical Inc. | $66.16 | +0.37% | +11.49% | +12.68% |
| CRNX | Crinetics Pharmaceuticals, Inc. | $84.86 | +1.20% | +81.60% | +182.02% |
| IMVT | Immunovant, Inc. | $43.50 | +8.51% | +67.89% | +192.14% |
| 4151.T | Kyowa Kirin Co., Ltd. | 2,596.00 JPY | −2.26% | +3.26% | +3.09% |
| RYTM | Rhythm Pharmaceuticals, Inc. | $113.35 | +6.80% | +7.94% | +14.26% |
| SRRK | Scholar Rock Holding Corporation | $58.25 | +19.05% | +36.10% | +74.77% |
| 0HI3.L | Arrowhead Pharmaceuticals, Inc. | $88.72 | +2.53% | +31.87% | +317.86% |
| ORKA | Oruka Therapeutics, Inc. | $101.82 | +12.88% | +260.17% | +601.72% |
| LQDA | Liquidia Corporation | $70.49 | −16.50% | +124.49% | +168.53% |
| KNSA | Kiniksa Pharmaceuticals International, plc | $80.20 | +26.23% | +90.93% | +142.74% |
| PTCT | PTC Therapeutics, Inc. Common Stock | $73.47 | −3.07% | −4.26% | +49.03% |
| DNTH | Dianthus Therapeutics, Inc. | $112.11 | +7.29% | +182.54% | +378.90% |
| TVTX | Travere Therapeutics, Inc. | $67.01 | +18.23% | +69.56% | +288.91% |
| MIRM | Mirum Pharmaceuticals, Inc. | $98.08 | −14.12% | +25.63% | +36.58% |
| GRFS | Grifols, S.A. | $8.09 | +4.39% | −12.45% | −17.62% |
| DYN | Dyne Therapeutics, Inc. | $26.34 | +2.77% | +42.38% | +102.62% |
| EWTX | Edgewise Therapeutics, Inc. | $44.21 | +6.27% | +86.15% | +209.16% |
| AMLX | Amylyx Pharmaceuticals, Inc. | $37.46 | +92.20% | +228.02% | +340.71% |
| MANE | Veradermics, Incorporated | $102.35 | −8.14% | +171.13% | +171.13% |
| CAMX.ST | Camurus AB (publ) | 609.50 SEK | −2.25% | −0.41% | −14.52% |
| CPRX | Catalyst Pharmaceuticals Inc | $31.49 | 0.00% | +36.03% | +52.72% |
| CLDX | Celldex Therapeutics, Inc. | $41.22 | +15.62% | +52.61% | +87.96% |
| IRON | Disc Medicine, Inc. | $82.41 | +10.45% | +4.20% | +38.71% |
| ARQT | Arcutis Biotherapeutics, Inc. | $24.86 | −10.70% | −14.28% | +58.44% |
| 0RD1.L | Camurus AB (publ) | 615.00 SEK | +0.61% | +0.29% | −13.50% |
| VRDN | Viridian Therapeutics, Inc. | $24.18 | +22.74% | −21.19% | +30.21% |
| PHVS | Pharvaris N.V. | $37.31 | +9.83% | +40.90% | +57.76% |
| RARE | Ultragenyx Pharmaceutical Inc. | $26.46 | −1.60% | +12.12% | −9.60% |
| TRVI | Trevi Therapeutics, Inc. | $17.95 | −0.94% | +50.97% | +146.57% |
| VERA | Vera Therapeutics, Inc. | $35.28 | +0.28% | −27.44% | +65.32% |
| NKTR | Nektar Therapeutics | $74.96 | +5.06% | +72.64% | +176.20% |
| BCRX | BioCryst Pharmaceuticals, Inc. | $9.83 | +2.61% | +31.07% | +17.16% |
| MLYS | Mineralys Therapeutics, Inc. | $26.34 | −3.20% | −26.24% | +78.70% |
| 009420.KS | Hanall Biopharma Co., Ltd. | 63,300.00 KRW | +40.04% | +50.00% | +108.57% |
| COAG | Hemab Therapeutics Holdings, Inc. | $49.65 | −1.47% | +46.03% | +46.03% |
| AUPH | Aurinia Pharmaceuticals Inc. | $16.75 | +10.05% | +9.12% | +38.03% |
| ZBIO | Zenas BioPharma, Inc. | $33.77 | +2.89% | −2.12% | +103.93% |
| STOK | Stoke Therapeutics, Inc. | $33.46 | +14.16% | +8.28% | +77.32% |
| ADMA | ADMA Biologics, Inc. | $9.72 | +14.62% | −45.64% | −42.86% |
| PVLA | Palvella Therapeutics, Inc. | $148.24 | +4.56% | +47.83% | +195.71% |
| NEU.AX | Neuren Pharmaceuticals Limited | 22.51 AUD | +32.02% | +19.04% | +18.47% |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $33.90 | −8.30% | +24.72% | −9.02% |
| SEPN | Septerna, Inc. | $41.30 | +23.32% | +47.61% | +245.32% |
| ETON | Eton Pharmaceuticals, Inc. | $63.05 | +49.02% | +287.76% | +268.93% |
| ANIP | ANI Pharmaceuticals, Inc. | $75.65 | −3.47% | −3.54% | −16.24% |
| ANAB | AnaptysBio, Inc. | $58.37 | +8.88% | +94.78% | +335.60% |
| AVLN | Avalyn Pharma Inc. | $38.20 | +38.31% | +29.54% | +29.54% |
| 0L35.L | Sarepta Therapeutics, Inc. | $18.94 | +3.95% | −11.24% | +4.63% |
| MAZE | Maze Therapeutics, Inc. | $30.42 | +6.03% | −23.51% | +128.55% |
| FBRX | Forte Biosciences, Inc. | $76.97 | +0.61% | +226.01% | +580.25% |
| TRAX | First Tracks Biotherapeutics, Inc. | $45.11 | +7.33% | +137.42% | +137.42% |
| XERS | Xeris Biopharma Holdings, Inc. | $8.74 | +6.46% | +18.91% | +12.48% |
| TYRA | Tyra Biosciences, Inc. | $26.69 | −14.07% | −0.37% | +122.42% |
| 4516.T | Nippon Shinyaku Co., Ltd. | 3,609.00 JPY | +0.81% | −34.02% | +18.84% |
| A2RR34.SA | Arrowhead Pharmaceuticals, Inc. | 58.02 BRL | +4.58% | +25.99% | +301.52% |
| PYC.AX | PYC Therapeutics Limited | 2.12 AUD | +30.46% | +28.10% | +63.71% |
| KALV | KalVista Pharmaceuticals Inc | $27.00 | 0.00% | +74.64% | +107.85% |
| 0LIF.L | Ultragenyx Pharmaceutical Inc. | $26.69 | +3.33% | +14.67% | −11.63% |
| OMER | Omeros Corporation | $19.28 | +70.02% | +17.70% | +343.22% |
| JBIO | Jade Biosciences, Inc. | $21.84 | +9.47% | +54.24% | +176.46% |
| MLTX | MoonLake Immunotherapeutics | $15.56 | −21.73% | +30.21% | −71.52% |
| 0HB0.L | Agios Pharmaceuticals, Inc. | $33.99 | +2.11% | +25.84% | −14.25% |
| SVRA | Savara Inc. | $5.60 | +3.13% | −0.71% | +78.34% |
| 006280.KS | GC Biopharma Corp. | 129,300.00 KRW | +7.21% | −18.07% | −1.88% |
| AVTX | Avalo Therapeutics, Inc. | $19.74 | +1.23% | +15.98% | +110.00% |
| WVE | Wave Life Sciences, Inc. | $5.25 | −11.32% | −67.08% | −47.18% |
| CLYM | Climb Bio Inc | $17.84 | +39.48% | +375.73% | +619.35% |
| DSGN | Design Therapeutics, Inc. | $15.68 | +31.99% | +72.31% | +201.54% |
| ANNX | Annexon, Inc. | $5.15 | +0.39% | +9.81% | +139.53% |
| DBVT | DBV Technologies S.A. | $14.97 | +7.70% | −21.42% | +52.91% |
| DBV.PA | DBV Technologies S.A. | 2.44 EUR | +2.78% | −25.33% | +54.11% |
| SLN | Silence Therapeutics plc | $13.67 | +13.26% | +122.28% | +170.16% |
| GNFT.PA | Genfit S.A. | 13.90 EUR | +5.30% | +164.76% | +293.99% |
| PHAR | Pharming Group N.V. | $11.46 | −11.93% | −33.14% | −19.75% |
| STTK | Shattuck Labs, Inc. | $8.06 | +14.49% | +117.25% | +710.87% |
| 0QNA.L | Basilea Pharmaceutica AG | 64.97 CHF | +1.43% | +18.64% | +38.97% |
| TECX | Tectonic Therapeutic, Inc. | $37.71 | +23.20% | +80.34% | +53.48% |
| IRWD | Ironwood Pharmaceuticals, Inc. | $4.39 | +12.28% | +2.81% | +245.67% |
| ASMB | Assembly Biosciences, Inc. | $35.33 | +36.36% | +4.68% | +32.72% |
| ZVRA | Zevra Therapeutics, Inc. | $12.04 | +24.00% | +44.36% | +28.63% |
| AGMB | AgomAb Therapeutics NV | $14.02 | +12.07% | −4.30% | −4.30% |
| GYRE | Gyre Therapeutics, Inc. | $6.68 | +8.97% | −1.91% | −18.54% |
| CRMD | CorMedix Inc. | $8.56 | +12.63% | −29.61% | −37.65% |
| AYJ.DE | Valneva SE | 2.86 EUR | +29.01% | −26.86% | −25.10% |
| VALN | Valneva SE | $6.78 | +36.69% | −24.29% | −28.10% |
| ZURA | Zura Bio Limited | $6.32 | +17.47% | +13.87% | +208.29% |
| AQST | Aquestive Therapeutics, Inc. | $5.08 | +36.19% | −21.24% | +31.61% |
| 0KBU.L | Omeros Corporation | $19.34 | −0.10% | +16.56% | +341.05% |
| SPRY | ARS Pharmaceuticals, Inc. | $5.72 | −0.69% | −50.22% | −57.66% |
| 005257.KS | GC Corp. | 16,630.00 KRW | +10.13% | −35.95% | −41.91% |
| KMDA | Kamada Ltd. Ordinary Shares | $8.63 | +23.46% | +26.48% | +26.12% |
| EPRX | Eupraxia Pharmaceuticals Inc. | $7.42 | +20.26% | −1.98% | +41.87% |
| EPRX.TO | Eupraxia Pharmaceuticals Inc. | 10.20 CAD | +16.70% | −2.49% | +41.67% |
| QTTB | Q32 Bio Inc. | $16.32 | +7.30% | +374.42% | +728.43% |
| RZLT | Rezolute, Inc. | $5.01 | +1.42% | +131.41% | −28.43% |
| AVIR | Atea Pharmaceuticals, Inc. | $5.43 | +12.66% | +56.03% | +48.36% |
| LRMR | Larimar Therapeutics, Inc. | $4.16 | +8.62% | +19.88% | +11.83% |
| XFOR | X4 Pharmaceuticals, Inc. | $4.16 | +5.58% | +6.94% | +4.79% |
| ENTA | Enanta Pharmaceuticals, Inc. | $14.08 | +10.60% | −2.36% | +62.59% |
| 4552.T | JCR Pharmaceuticals Co., Ltd. | 545.00 JPY | +5.21% | −19.79% | −7.40% |
| UPB | Upstream Bio, Inc. | $7.18 | +12.19% | −73.48% | −57.08% |
| CUV.AX | Clinuvel Pharmaceuticals Limited | 10.54 AUD | +7.44% | −17.07% | −21.84% |
| IKT | Inhibikase Therapeutics, Inc. | $2.50 | +6.38% | +28.21% | +42.05% |
| PRQR | ProQR Therapeutics N.V. | $2.38 | +37.57% | +19.60% | +5.78% |
| IFRX | InflaRx N.V. | $2.46 | +44.71% | +125.69% | +167.10% |
| TRDA | Entrada Therapeutics, Inc. | $7.62 | +25.33% | −24.18% | +35.35% |
| TTRX | Turn Therapeutics Inc. | $9.73 | +25.06% | +107.46% | +39.00% |
| FULC | Fulcrum Therapeutics, Inc. | $3.86 | +3.49% | −64.65% | −39.78% |
| SION | Sionna Therapeutics, Inc. | $6.25 | −86.23% | −84.06% | −74.02% |
| ELDN | Eledon Pharmaceuticals, Inc. | $3.47 | −2.80% | +114.20% | +35.02% |
| CAMP | CAMP4 THERAPEUTICS CORPORATION | $4.45 | +30.12% | −25.83% | +185.26% |
| IVVD | Invivyd, Inc. | $0.94 | +53.89% | −62.45% | +67.62% |
| 0OB3.L | Valneva SE | 2.87 EUR | +0.60% | −24.07% | −24.84% |
| AKBA | Akebia Therapeutics, Inc. | $0.96 | −25.12% | −38.17% | −68.37% |
| SLGL | Sol-Gel Technologies Ltd. | $81.72 | +2.37% | +93.88% | +289.14% |
| XUP.DE | Genfit S.A. | 14.08 EUR | −1.81% | +164.41% | +166.82% |
| 0QN1.L | Santhera Pharmaceuticals Holding AG | 15.70 CHF | −1.63% | +29.32% | +12.30% |
| 0T6G.L | Q32 Bio Inc. | $16.27 | +4.95% | — | — |
| RNA | Prosensa Holding B.V. Ordinary Shares | $14.00 | +21.00% | −5.08% | −5.08% |
| PEPG | PepGen Inc. | $3.43 | +80.26% | −52.23% | +161.45% |
| CRDL | Cardiol Therapeutics Inc. | $2.00 | +73.16% | +101.21% | +75.44% |
| CRDL.TO | Cardiol Therapeutics Inc. | 2.76 CAD | +68.29% | +102.94% | +72.50% |
| KROS | Keros Therapeutics, Inc. | $11.68 | +14.40% | −37.00% | −20.87% |
| ANTX | AN2 Therapeutics, Inc. | $5.66 | +14.81% | +409.91% | +403.11% |
| SIGA | SIGA Technologies, Inc. | $3.04 | −1.94% | −45.19% | −60.50% |
| ARMP | Armata Pharmaceuticals, Inc. | $5.56 | +7.96% | −15.24% | +113.85% |
| MSLE | Satellos Bioscience Inc. | $9.40 | −0.58% | +51.52% | +82.17% |
| MSCL.TO | Satellos Bioscience Inc. | 13.00 CAD | −2.26% | +54.76% | +80.56% |
| PLX | Protalix BioTherapeutics, Inc. | $2.41 | +0.42% | +38.51% | +50.62% |
| 0AB6.L | Hyloris Pharmaceuticals SA | 4.46 EUR | −0.66% | −37.71% | −32.01% |
| SPRB | Spruce Biosciences, Inc. | $63.40 | +50.99% | −30.10% | +697.48% |
| MDWD | MediWound Ltd. | $12.97 | −10.24% | −29.59% | −24.81% |
| AARD | Aardvark Therapeutics, Inc. | $7.25 | +8.37% | −45.03% | −19.80% |
| CRNO-B.ST | Cereno Scientific AB (publ) | 4.60 SEK | −4.52% | −33.57% | −53.95% |
| UNCY | Unicycive Therapeutics, Inc. | $5.47 | +6.63% | −5.69% | +27.51% |
| MIST | Milestone Pharmaceuticals Inc. | $1.16 | +2.65% | −43.41% | −31.36% |
| 4599.T | StemRIM | 357.00 JPY | +22.26% | +34.72% | +15.91% |
| EQ | Equillium, Inc. | $2.18 | −11.38% | +42.48% | +14.74% |
| DXB.AX | Dimerix Limited | 0.31 AUD | +22.00% | −44.55% | −35.79% |
| CPIX | Cumberland Pharmaceuticals Inc. | $8.70 | +59.59% | +118.59% | +166.87% |
| INO | Inovio Pharmaceuticals, Inc. | $1.29 | +32.99% | −23.67% | −33.85% |
| 0H8T.L | Aclaris Therapeutics, Inc. | $6.58 | +6.50% | +120.68% | +265.21% |
| MDP.TO | Medexus Pharmaceuticals Inc. | 5.15 CAD | +3.83% | +66.67% | +80.70% |
| RFL | Rafael Holdings, Inc. | $2.24 | −1.75% | +77.78% | +52.38% |
| PTHS | Pelthos Therapeutics Inc. | $27.20 | −1.41% | −7.64% | +20.41% |
| PYPD | PolyPid Ltd. | $5.10 | +28.14% | +17.24% | +47.83% |
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.