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Rare disease stocks
We cover 122 companies grouped under the Rare disease theme. The median market value in the group is $95.04M. Across the group, the median move over the past trading day is −0.13%. 50% of the 117 with a 200-day average trade above it. Price change over the past year runs from −82.9% to +170.6% across the middle 80 percent of the group, with a median of −8.1%.
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Table
122 of 268 companies match these filters.Clear filters
| Ticker | Company | Market cap (USD) | Size | 1Y return |
|---|---|---|---|---|
| VX1.DE | Vertex Pharmaceuticals Incorporated | $139.82B | Large cap | +41.64% |
| VRTX | Vertex Pharmaceuticals Incorporated | $138.63B | Large cap | +40.66% |
| DUL.DE | Alnylam Pharmaceuticals, Inc. | $32.28B | Large cap | −10.21% |
| ALNY | Alnylam Pharmaceuticals, Inc. | $32.12B | Large cap | −47.38% |
| A1LN34.SA | Alnylam Pharmaceuticals, Inc. | $31.78B | Large cap | −49.35% |
| INSM | Insmed Incorporated | $27.16B | Large cap | −7.54% |
| ROIV | Roivant Sciences Ltd. | $26.55B | Large cap | +206.51% |
| JAZZ | Jazz Pharmaceuticals plc | $16.89B | Large cap | +100.35% |
| ASND | Ascendis Pharma A/S | $16.41B | Large cap | +29.66% |
| BBIO | BridgeBio Pharma, Inc. | $15.52B | Large cap | +60.05% |
| CORT | Corcept Therapeutics Incorporated | $12.95B | Large cap | +71.81% |
| 0JAV.L | Insmed Incorporated | $12.89B | Large cap | −7.52% |
| BMRN | BioMarin Pharmaceutical Inc. | $12.70B | Large cap | +11.85% |
| REC.MI | Recordati Industria Chimica e Farmaceutica S.p.A. | $12.57B | Large cap | +1.80% |
| ARWR | Arrowhead Pharmaceuticals, Inc. | $12.52B | Large cap | +308.99% |
| CYTK | Cytokinetics, Incorporated | $11.01B | Large cap | +109.36% |
| IONS | Ionis Pharmaceuticals, Inc. | $10.56B | Large cap | +48.21% |
| KYMR | Kymera Therapeutics, Inc. | $10.53B | Large cap | +197.09% |
| 0JDI.L | Ionis Pharmaceuticals, Inc. | $10.40B | Large cap | +47.26% |
| ABVX.PA | ABIVAX Société Anonyme | $10.21B | Large cap | +48.91% |
| PTGX | Protagonist Therapeutics, Inc. | $9.80B | Mid cap | +146.62% |
| IMVT | Immunovant, Inc. | $9.07B | Mid cap | +189.13% |
| 4151.T | Kyowa Kirin Co., Ltd. | $8.51B | Mid cap | +4.01% |
| RYTM | Rhythm Pharmaceuticals, Inc. | $7.78B | Mid cap | +9.35% |
| SRRK | Scholar Rock Holding Corporation | $7.31B | Mid cap | +74.18% |
| 0HI3.L | Arrowhead Pharmaceuticals, Inc. | $6.98B | Mid cap | +294.59% |
| ORKA | Oruka Therapeutics, Inc. | $6.58B | Mid cap | +555.07% |
| LQDA | Liquidia Corporation | $6.31B | Mid cap | +154.86% |
| TVTX | Travere Therapeutics, Inc. | $6.24B | Mid cap | +284.86% |
| DNTH | Dianthus Therapeutics, Inc. | $6.13B | Mid cap | +356.93% |
| PTCT | PTC Therapeutics, Inc. Common Stock | $6.07B | Mid cap | +46.02% |
| KNSA | Kiniksa Pharmaceuticals International, plc | $6.05B | Mid cap | +133.47% |
| MIRM | Mirum Pharmaceuticals, Inc. | $5.98B | Mid cap | +31.96% |
| GRFS | Grifols, S.A. | $5.38B | Mid cap | −19.39% |
| DYN | Dyne Therapeutics, Inc. | $4.92B | Mid cap | +93.26% |
| EWTX | Edgewise Therapeutics, Inc. | $4.80B | Mid cap | +207.13% |
| MANE | Veradermics, Incorporated | $4.29B | Mid cap | +172.32% |
| CAMX.ST | Camurus AB (publ) | $3.79B | Mid cap | −15.35% |
| CLDX | Celldex Therapeutics, Inc. | $3.19B | Mid cap | +79.51% |
| IRON | Disc Medicine, Inc. | $3.17B | Mid cap | +37.90% |
| ARQT | Arcutis Biotherapeutics, Inc. | $3.12B | Mid cap | +60.64% |
| 0RD1.L | Camurus AB (publ) | $2.84B | Mid cap | −15.10% |
| VRDN | Viridian Therapeutics, Inc. | $2.75B | Mid cap | +30.71% |
| VERA | Vera Therapeutics, Inc. | $2.65B | Mid cap | +67.21% |
| RARE | Ultragenyx Pharmaceutical Inc. | $2.61B | Mid cap | −12.10% |
| PHVS | Pharvaris N.V. | $2.60B | Mid cap | +55.59% |
| TRVI | Trevi Therapeutics, Inc. | $2.55B | Mid cap | +124.00% |
| NKTR | Nektar Therapeutics | $2.52B | Mid cap | +151.95% |
| BCRX | BioCryst Pharmaceuticals, Inc. | $2.51B | Mid cap | +15.38% |
| 009420.KS | Hanall Biopharma Co., Ltd. | $2.36B | Mid cap | +112.62% |
| COAG | Hemab Therapeutics Holdings, Inc. | $2.30B | Mid cap | +44.68% |
| MLYS | Mineralys Therapeutics, Inc. | $2.28B | Mid cap | +70.22% |
| PVLA | Palvella Therapeutics, Inc. | $2.22B | Mid cap | +173.39% |
| AUPH | Aurinia Pharmaceuticals Inc. | $2.20B | Mid cap | +36.58% |
| ADMA | ADMA Biologics, Inc. | $2.16B | Mid cap | −44.17% |
| ZBIO | Zenas BioPharma, Inc. | $2.15B | Mid cap | +97.73% |
| STOK | Stoke Therapeutics, Inc. | $2.14B | Mid cap | +71.45% |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $2.03B | Mid cap | −13.45% |
| ABNX.PA | ABIONYX Pharma SA | $98.59M | Micro cap | −31.34% |
| 0RAG.L | ABIONYX Pharma SA | $95.77M | Micro cap | −29.10% |
| ILA.AX | Island Pharmaceuticals Limited | $95.70M | Micro cap | +109.30% |
| FBIO | Fortress Biotech, Inc. | $94.38M | Micro cap | +20.09% |
| PLYX | Polaryx Therapeutics, Inc. | $94.21M | Micro cap | −94.03% |
| PAR.AX | Paradigm Biopharmaceuticals Limited | $92.46M | Micro cap | −24.56% |
| RLYB | Rallybio Corporation | $89.64M | Micro cap | +257.63% |
| GOSS | Gossamer Bio, Inc. | $85.01M | Micro cap | −92.93% |
| RCE.AX | Recce Pharmaceuticals Ltd | $82.40M | Micro cap | −10.71% |
| TELO | Telomir Pharmaceuticals, Inc. | $80.47M | Micro cap | −20.95% |
| STX.L | Shield Therapeutics plc | $71.97M | Micro cap | −37.42% |
| FBIOP | Fortress Biotech, Inc. | $71.45M | Micro cap | +131.74% |
| ALTHX.PA | THX Pharma Société Anonyme | $71.22M | Micro cap | +73.96% |
| SPRO | Spero Therapeutics, Inc. | $71.05M | Micro cap | −38.38% |
| RNTX | Rein Therapeutics Inc. | $69.01M | Micro cap | −27.27% |
| IMA | ImageneBio, Inc. | $61.64M | Micro cap | −60.19% |
| SCYX | SCYNEXIS, Inc. | $56.41M | Micro cap | +579.04% |
| ATYR | aTyr Pharma, Inc. | $54.05M | Micro cap | −89.34% |
| NSRX | Nasus Pharma Ltd. | $48.01M | Micro cap | −49.75% |
| MCRB | Seres Therapeutics, Inc. | $47.81M | Micro cap | −74.79% |
| EDSA | Edesa Biotech, Inc. | $47.63M | Micro cap | +105.83% |
| GRCE | Grace Therapeutics, Inc. | $46.28M | Micro cap | −29.97% |
| MREO | Mereo BioPharma Group plc | $44.39M | Micro cap | −84.20% |
| BOT.AX | Botanix Pharmaceuticals Limited | $41.30M | Micro cap | −86.67% |
| NNVC | NanoViricides, Inc. | $32.68M | Micro cap | −9.86% |
| QNCX | Quince Therapeutics, Inc. | $31.84M | Micro cap | +100.71% |
| IMM.L | ImmuPharma plc | $31.75M | Micro cap | +89.66% |
| 520A.T | J-PHARMACEUTICALS CO LTD | $31.71M | Micro cap | −60.00% |
| ALDVI.PA | Advicenne S.A. | $30.93M | Micro cap | +39.11% |
| 4591.T | Ribomic Inc. | $30.52M | Micro cap | −17.31% |
| BRNS | Barinthus Biotherapeutics plc | $28.86M | Micro cap | −38.04% |
| 0RQJ.L | Saniona AB (publ) | $27.98M | Micro cap | −12.20% |
| RVX.TO | Resverlogix Corp. | $24.81M | Micro cap | 0.00% |
| JSPR | Jasper Therapeutics, Inc. | $23.96M | Micro cap | −75.24% |
| TVRD | Tvardi Therapeutics, Inc. | $23.74M | Micro cap | −94.18% |
| 4884.T | Kringle Pharma, Inc. | $22.30M | Micro cap | −11.29% |
| COCP | Cocrystal Pharma, Inc. | $21.57M | Micro cap | −27.74% |
| KTTA | Pasithea Therapeutics Corp. | $19.05M | Micro cap | −24.30% |
| CALC | CalciMedica, Inc. | $18.06M | Micro cap | −79.74% |
| 4881.T | FunPep Company Limited | $15.92M | Micro cap | −55.65% |
| HIND | Vyome Holdings, Inc. | $15.16M | Micro cap | −79.68% |
| ALBPS.PA | Biophytis S.A. | $11.98M | Micro cap | −50.31% |
| AZTR | Azitra, Inc. | $10.48M | Micro cap | −83.21% |
| QNRX | Quoin Pharmaceuticals, Ltd. | $10.40M | Micro cap | −29.81% |
| ANNX.ST | Annexin Pharmaceuticals AB (publ) | $10.27M | Micro cap | −46.03% |
| PHR.WA | Pharmena S.A. | $9.45M | Micro cap | −8.72% |
| IMRN | Immuron Limited | $9.20M | Micro cap | −35.77% |
| IMC.AX | Immuron Limited | $8.58M | Micro cap | −46.38% |
| SNGX | Soligenix, Inc. | $8.36M | Micro cap | −87.20% |
| XCUR | Exicure, Inc. | $8.25M | Micro cap | −77.93% |
| INM | InMed Pharmaceuticals Inc. | $7.64M | Micro cap | −36.44% |
| VBS.AX | Vectus Biosystems Limited | $6.65M | Micro cap | +104.82% |
| PULM | Pulmatrix, Inc. | $5.57M | Micro cap | −68.62% |
| BIOSGN.ST | Biosergen AB (publ) | $5.31M | Micro cap | −94.62% |
| JAGX | Jaguar Health, Inc. | $4.78M | Micro cap | −98.52% |
| GRI | GRI Bio, Inc. | $4.28M | Micro cap | −95.23% |
| XRTX | XORTX Therapeutics Inc. | $4.03M | Micro cap | −43.99% |
| BIT.AX | Biotron Limited | $3.99M | Micro cap | −50.00% |
| SXTP | 60 Degrees Pharmaceuticals, Inc. | $3.80M | Micro cap | −78.23% |
| MTNB | Matinas BioPharma Holdings, Inc. | $2.89M | Micro cap | −90.08% |
| APTA.ST | Aptahem AB (publ) | $1.50M | Micro cap | −67.65% |
| APLI.TO | Appili Therapeutics Inc. | $1.44M | Micro cap | −25.00% |
| VBIO | Valion Bio, Inc. | $647.60K | Micro cap | −94.92% |
| ALNEV.PA | Neovacs S.A. | $22.00 | Micro cap | −11.29% |
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.