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Rare disease stocks
We cover 99 companies grouped under the Rare disease theme. The median market value in the group is $2.20B. Across the group, the median move over the past trading day is −0.30%. 54% of the 96 with a 200-day average trade above it. Price change over the past year runs from −84.8% to +206.6% across the middle 80 percent of the group, with a median of +20.1%.
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Table
99 of 268 companies match these filters.Clear filters
| Ticker | Company | Price | 1M return | YTD return | 1Y return |
|---|---|---|---|---|---|
| VRTX34.SA | Vertex Pharmaceuticals Incorporated | 88.56 BRL | +14.82% | −85.60% | −83.18% |
| VRTX | Vertex Pharmaceuticals Incorporated | $547.29 | +11.60% | +21.05% | +40.66% |
| ARGX | argenx SE | $1,047.10 | +20.05% | +24.66% | +47.90% |
| A1RG34.SA | argenx SE | 209.16 BRL | +14.71% | +11.37% | +36.97% |
| ALNY | Alnylam Pharmaceuticals, Inc. | $239.00 | −17.13% | −40.28% | −47.38% |
| A1LN34.SA | Alnylam Pharmaceuticals, Inc. | 62.25 BRL | −15.31% | −44.14% | −49.35% |
| INSM | Insmed Incorporated | $124.39 | +19.46% | −29.77% | −7.54% |
| ROIV | Roivant Sciences Ltd. | $36.75 | +8.60% | +67.58% | +206.51% |
| JAZZ | Jazz Pharmaceuticals plc | $249.29 | −2.97% | +43.97% | +100.35% |
| ASND | Ascendis Pharma A/S | $250.25 | −1.65% | +17.33% | +29.66% |
| BBIO | BridgeBio Pharma, Inc. | $79.37 | −7.22% | +1.45% | +60.05% |
| CORT | Corcept Therapeutics Incorporated | $119.80 | +27.19% | +213.61% | +71.81% |
| BMRN | BioMarin Pharmaceutical Inc. | $65.61 | +5.70% | +10.36% | +11.85% |
| B1MR34.SA | BioMarin Pharmaceutical Inc. | 166.77 BRL | +12.08% | −0.14% | +4.97% |
| ARWR | Arrowhead Pharmaceuticals, Inc. | $89.12 | +3.89% | +31.46% | +308.99% |
| CYTK | Cytokinetics, Incorporated | $79.20 | −0.31% | +28.30% | +109.36% |
| IONS | Ionis Pharmaceuticals, Inc. | $63.36 | +14.58% | −20.47% | +48.21% |
| KYMR | Kymera Therapeutics, Inc. | $125.55 | +18.26% | +72.55% | +197.09% |
| PTGX | Protagonist Therapeutics, Inc. | $151.50 | +10.71% | +73.78% | +146.62% |
| IMVT | Immunovant, Inc. | $43.89 | +12.83% | +69.39% | +189.13% |
| CRNX | Crinetics Pharmaceuticals, Inc. | $84.84 | +1.17% | +81.55% | +179.08% |
| RYTM | Rhythm Pharmaceuticals, Inc. | $111.87 | +3.54% | +6.53% | +9.35% |
| SRRK | Scholar Rock Holding Corporation | $60.04 | +21.61% | +40.28% | +74.18% |
| ORKA | Oruka Therapeutics, Inc. | $99.44 | +9.94% | +251.75% | +555.07% |
| LQDA | Liquidia Corporation | $71.36 | −17.81% | +127.26% | +154.86% |
| TVTX | Travere Therapeutics, Inc. | $67.12 | +20.05% | +69.84% | +284.86% |
| DNTH | Dianthus Therapeutics, Inc. | $110.76 | +9.01% | +179.13% | +356.93% |
| PTCT | PTC Therapeutics, Inc. Common Stock | $72.69 | −3.58% | −5.28% | +46.02% |
| KNSA | Kiniksa Pharmaceuticals International, plc | $78.68 | −0.96% | +87.31% | +133.47% |
| MIRM | Mirum Pharmaceuticals, Inc. | $98.51 | −12.22% | +26.18% | +31.96% |
| GRFS | Grifols, S.A. | $7.90 | −1.25% | −14.50% | −19.39% |
| DYN | Dyne Therapeutics, Inc. | $26.36 | +1.78% | +42.49% | +93.26% |
| EWTX | Edgewise Therapeutics, Inc. | $44.38 | +9.99% | +86.86% | +207.13% |
| MANE | Veradermics, Incorporated | $102.80 | −7.84% | +172.32% | +172.32% |
| AMLX | Amylyx Pharmaceuticals, Inc. | $37.08 | +89.67% | +224.69% | +292.38% |
| CPRX | Catalyst Pharmaceuticals Inc | $31.49 | 0.00% | +36.03% | +55.97% |
| CLDX | Celldex Therapeutics, Inc. | $40.57 | +14.02% | +50.20% | +79.51% |
| IRON | Disc Medicine, Inc. | $82.67 | +10.74% | +4.53% | +37.90% |
| ARQT | Arcutis Biotherapeutics, Inc. | $25.14 | −11.54% | −13.31% | +60.64% |
| VRDN | Viridian Therapeutics, Inc. | $24.26 | +24.35% | −20.93% | +30.71% |
| VERA | Vera Therapeutics, Inc. | $36.82 | +10.50% | −24.27% | +67.21% |
| RARE | Ultragenyx Pharmaceutical Inc. | $26.44 | +1.77% | +12.03% | −12.10% |
| PHVS | Pharvaris N.V. | $37.00 | +9.97% | +39.73% | +55.59% |
| TRVI | Trevi Therapeutics, Inc. | $17.92 | −2.77% | +50.71% | +124.00% |
| NKTR | Nektar Therapeutics | $73.77 | +4.24% | +69.90% | +151.95% |
| BCRX | BioCryst Pharmaceuticals, Inc. | $9.83 | +0.92% | +31.07% | +15.38% |
| COAG | Hemab Therapeutics Holdings, Inc. | $49.19 | +4.08% | +44.68% | +44.68% |
| MLYS | Mineralys Therapeutics, Inc. | $25.89 | −3.93% | −27.50% | +70.22% |
| PVLA | Palvella Therapeutics, Inc. | $154.33 | +8.04% | +53.90% | +173.39% |
| AUPH | Aurinia Pharmaceuticals Inc. | $16.54 | +10.56% | +7.75% | +36.58% |
| ADMA | ADMA Biologics, Inc. | $9.63 | +9.56% | −46.14% | −44.17% |
| ZBIO | Zenas BioPharma, Inc. | $33.04 | +2.32% | −4.23% | +97.73% |
| STOK | Stoke Therapeutics, Inc. | $33.09 | +12.59% | +7.09% | +71.45% |
| AGIO | Agios Pharmaceuticals, Inc. Common Stock | $33.99 | −5.56% | +25.06% | −13.45% |
| FBIO | Fortress Biotech, Inc. | $2.81 | −17.11% | −30.27% | +20.09% |
| PLYX | Polaryx Therapeutics, Inc. | $2.09 | +5.03% | −94.03% | −94.03% |
| RLYB | Rallybio Corporation | $16.88 | +4.49% | +198.44% | +257.63% |
| GOSS | Gossamer Bio, Inc. | $0.17 | −15.17% | −93.96% | −92.93% |
| TELO | Telomir Pharmaceuticals, Inc. | $1.17 | −0.85% | −12.69% | −20.95% |
| TENX | Tenax Therapeutics, Inc. | $2.12 | −86.58% | −80.81% | −64.19% |
| FBIOP | Fortress Biotech, Inc. | $18.40 | −1.92% | +81.10% | +131.74% |
| SPRO | Spero Therapeutics, Inc. | $1.22 | +2.09% | −48.09% | −38.38% |
| RNTX | Rein Therapeutics Inc. | $0.80 | +2.30% | −36.00% | −27.27% |
| IMA | ImageneBio, Inc. | $5.43 | −5.73% | −19.67% | −60.19% |
| SCYX | SCYNEXIS, Inc. | $5.67 | +56.20% | +787.32% | +579.04% |
| ATYR | aTyr Pharma, Inc. | $0.55 | +11.54% | −27.40% | −89.34% |
| NSRX | Nasus Pharma Ltd. | $4.05 | +33.66% | −46.36% | −49.75% |
| MCRB | Seres Therapeutics, Inc. | $4.76 | −0.83% | −68.58% | −74.79% |
| EDSA | Edesa Biotech, Inc. | $4.94 | −0.60% | +229.33% | +105.83% |
| GRCE | Grace Therapeutics, Inc. | $2.15 | −4.44% | −41.26% | −29.97% |
| MREO | Mereo BioPharma Group plc | $0.28 | −10.03% | −45.49% | −84.20% |
| NNVC | NanoViricides, Inc. | $1.28 | −20.50% | +5.79% | −9.86% |
| QNCX | Quince Therapeutics, Inc. | $31.31 | +61.64% | +4.37% | +100.71% |
| BRNS | Barinthus Biotherapeutics plc | $0.71 | +23.93% | −3.89% | −38.04% |
| RVX.TO | Resverlogix Corp. | 0.10 CAD | 0.00% | 0.00% | 0.00% |
| JSPR | Jasper Therapeutics, Inc. | $0.72 | +13.43% | −60.98% | −75.24% |
| TVRD | Tvardi Therapeutics, Inc. | $1.78 | −27.05% | −56.37% | −94.18% |
| COCP | Cocrystal Pharma, Inc. | $1.12 | +20.43% | +13.13% | −27.74% |
| VYNE | VYNE Therapeutics Inc | $28.56 | +3080.40% | +4824.14% | +8660.74% |
| KTTA | Pasithea Therapeutics Corp. | $0.57 | +26.67% | −52.10% | −24.30% |
| CALC | CalciMedica, Inc. | $0.59 | −18.18% | −91.28% | −79.74% |
| CMMB | Chemomab Therapeutics Ltd. | $2.59 | +81.12% | +44.69% | −25.57% |
| HIND | Vyome Holdings, Inc. | $2.16 | +8.54% | −39.15% | −79.68% |
| AZTR | Azitra, Inc. | $0.17 | +33.00% | −38.03% | −83.21% |
| QNRX | Quoin Pharmaceuticals, Ltd. | $5.18 | +23.33% | −63.21% | −29.81% |
| IMRN | Immuron Limited | $1.14 | +4.11% | +43.40% | −35.77% |
| SNGX | Soligenix, Inc. | $0.38 | +6.67% | −72.96% | −87.20% |
| XCUR | Exicure, Inc. | $1.26 | −27.59% | −77.42% | −77.93% |
| INM | InMed Pharmaceuticals Inc. | $1.43 | −14.37% | +27.68% | −36.44% |
| PULM | Pulmatrix, Inc. | $1.53 | −9.23% | −32.52% | −68.62% |
| CVKD | Cadrenal Therapeutics, Inc. | $1.55 | −22.89% | −78.85% | −88.87% |
| JAGX | Jaguar Health, Inc. | $0.92 | −4.63% | −97.37% | −98.52% |
| GLMD | Galmed Pharmaceuticals Ltd. | $4.33 | +710.86% | +426.12% | +211.51% |
| GRI | GRI Bio, Inc. | $1.91 | +33.57% | −75.64% | −95.23% |
| XRTX | XORTX Therapeutics Inc. | $2.22 | +8.20% | −22.18% | −43.99% |
| SXTP | 60 Degrees Pharmaceuticals, Inc. | $1.08 | −28.00% | −47.67% | −78.23% |
| MTNB | Matinas BioPharma Holdings, Inc. | $0.20 | −32.97% | −68.32% | −90.08% |
| APLI.TO | Appili Therapeutics Inc. | 0.02 CAD | +50.00% | −40.00% | −25.00% |
| VBIO | Valion Bio, Inc. | $0.16 | −45.83% | −89.01% | −94.92% |
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.